Plenary 1: Hope for All: Addressing the Needs of Those with Untreated CF Mutations

Presentation Date: Thursday, November 3, 2022

As most people with CF who have two nonsense or other rare mutations cannot benefit from CFTR modulators, there is a critical need to develop treatments that target the underlying cause of their disease. Plenary 1 will provide an overview of less common mutations, such as nonsense mutations and splice variants, and the different scientific approaches being undertaken to develop new treatments. Steven M. Rowe, MD, Chief Scientific Officer at the Cystic Fibrosis Foundation, will focus on nonsense mutations and the innovative new research underway to help enable the production of a functioning CFTR protein. Michelle Hastings, PhD, Rosalind Franklin School of Medicine and Science, will highlight research into the development of a novel approach using antisense oligonucleotides (ASOs) for individuals with splice mutations. Rebecca Darrah, PhD, Case Western Reserve University, a geneticist as well as a genetic counselor, will delve into other rare mutations and the latest research into mRNA therapy which may prove to be a viable new treatment for people with these and other mutations.

Educational Objectives

• Describe rare CFTR variants and how these mutations are more challenging to treat with current CFTR modulators.
• Demonstrate how progress in overcoming nonsense mutations through mechanisms that induce translational readthrough have the potential to lead to novel treatment strategies.
• Understand how mutations can cause an array of CF manifestations but may be amenable to nucleotide therapies, such as antisense oligonucleotides .
• Describe how mRNA therapy has the potential to restore CFTR function to the lung, regardless of underlying CFTR mutation.

Plenary 2: It Takes Everyone: Novel CF Therapeutics & Clinical Trial Strategy to Accelerate Our Mission

Presentation Date: Friday, November 4, 2022

Significant progress has been made in the last two decades to advance therapies for people with CF, transforming the lives of many individuals with the disease. However, there is still a critical need for innovative new therapies to treat the underlying cause of CF as well as the many complications of the disease. Plenary 2 will discuss clinical trial advances in the CF therapeutics pipeline and highlight the diverse strategies being implemented to address the needs of all people with CF. Deepika Polineni, MD, MPH, will review past and current drug trials that are targeting the needs of people with CF via a multi-organ approach. Nicole Mayer Hamblett, PhD, will provide an overview of the comprehensive clinical trial strategy to enable novel therapeutics and optimal treatment regimens to quickly advance, furthering our mission of ensuring all with CF will live long, healthy lives.

Educational Objectives:

• Describe the recent advances in potential therapies focused on correcting the underlying defect in cystic fibrosis.
• Describe the types of clinical trials in our pipeline supporting a population with CF with different needs and until curative disease-modifying therapies are realized for all.
• Demonstrate the impact of trial participation on advancing the entire therapeutic pipeline through novel study designs and the unification of data.

Plenary 3: Evolving with the Times: Staying Agile with a Diverse and Aging CF Patient Population

Better health and longer survival of those living with cystic fibrosis compel us to adapt how we partner with our patients and their families to provide quality CF care. Shifts in what CF care teams are doing now and what we invest in to support the continued evolution to improve care coordination and disease management is what Elizabeth Tullis, MD, FRCPC, will walk us through in this year’s third plenary.

Educational Objectives:

• Identify the key drivers in the changes in CF care
• Describe areas of focus to improve CF care
• Recognize roles and efforts to propel us forward at the clinic and community level