Plenary 1: Genetic Therapies for All: Harnessing Cross-Disease Knowledge for Breakthroughs in Cystic Fibrosis

Presentation Date: Thursday, November 2, 2023

In this plenary session, experts will explore how we can gain valuable insights from the development of genetic therapies for other diseases and apply these approaches to cystic fibrosis. Alexis Thompson, MD, will discuss how gene therapy has advanced in sickle cell disease and other genetic blood disorders, how the science has evolved, challenges they encountered and new ones they still face, and how the community has been a critical partner in the successful development of therapies. Paul McCray, MD, will build on Dr. Thompson’s talk to explain the unique challenges of developing a genetic therapy in CF, how we might overcome these obstacles, and recent advances that represent foundational progress. This comprehensive session will pave the way for improved understanding of how we will develop novel genetic therapies which could benefit all people with CF, regardless of their mutations.

Learning Objectives:

• Discuss how genetic therapies have advanced for other blood disorders/diseases such as sickle cell disease.
• Identify challenges and approaches to implementing genetic therapy in patients.
• Describe how gene therapy has the potential to restore CFTR function to the lung, regardless of the underlying CFTR mutation.
• Understand the challenges of developing genetic therapies for cystic fibrosis and how this differs from blood disorders.

Plenary 2: "Micro-Management": The Changing Face of Infections in CF

Presentation Date: Friday, November 3, 2023

Significant advancements in the treatment of cystic fibrosis over the past several decades have greatly improved the lives of people with CF. However, lung infections remain a persistent complication of the disease, and there continues to be a critical need to improve the diagnosis and treatment of lung infections and inflammation in all people with CF. Plenary 2 will focus on the current landscape of CF infections, including the latest clinical research. Luke Hoffman, MD, PhD, will provide an overview of infections in CF, how they are changing in the context of highly effective modulators, and what challenges are emerging. Natalie E. West, MD, MHS, will focus on the development of new therapeutics in CF and describe novel approaches to managing CF infections with the goal of improving the lives of all people with CF.

Learning Objectives:

• Describe how infections in CF are changing in the era of CFTR modulators.

• Identify the beneficial and adverse effects of antibiotics in CF care.
• Recognize how our approach to treatment of infection is changing and highlight clinical trials in the pipeline.
• Highlight ongoing efforts to re-evaluate and improve our approach to infection diagnosis and treatment.

Plenary 3: There is No Health Without Mental Health: Progress, Challenges, and Hope for the Future

Presentation Date: Saturday, November 4, 2023

For all of us, mental health is critical for overall health. Plenary 3 will focus on the integration of mental health into CF care and research. Beth Smith, MD, will describe the foundation for clinical care practices made possible through partnerships between people with CF, their families, and care teams. Anna Georgiopoulos, MD, will provide an overview of research—including a pipeline of evidence-based interventions—and priorities for the future. Lastly, Shaina Blair, MSW, LCSW, will highlight how the mental health and wellness of CF care teams is connected to sustaining quality care for patients and families.

Learning Objectives:

• Identify how the international guidelines for screening and treating depression and anxiety in people with CF are being implemented.

• Describe recent advances and priorities for future research in CF mental health.
• Recognize factors influencing well-being for members of CF care teams and how it can affect quality of care.