NACFC 2023 | Genetic Therapies for All: Harnessing Cross-Disease Knowledge for Breakthroughs in CF

During plenary 1 of our North American Cystic Fibrosis Conference, Paul McCray, Jr., MD, and Alexis Thompson, MD, share how we can gain valuable insights from the development of genetic therapies for other diseases and apply these approaches to cystic fibrosis.

Dr. Thompson discusses how gene therapy has advanced in sickle cell disease and other genetic blood disorders, how the science has evolved, the challenges encountered and new ones they still face, and how the community has been a critical partner in the successful development of therapies. Dr. McCray builds 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 paves the way for an improved understanding of how we will develop novel genetic therapies that could benefit all people with CF, regardless of their mutations.

0:00:00 | Genetic Therapies for All: Harnessing Cross-Disease Knowledge for Breakthroughs in CF
00:19 | CF Foundation President and CEO Michael Boyle, MD, kicks off the conference and welcomes viewers to Plenary 1.
07:13 | KC White, chair of the CF Foundation Board of Trustees, shares a message of gratitude and hope for the future.
12:44 | Steven Rowe, MD, chief scientific officer of the CF Foundation, shares recent research funding highlights and introduces Alexis Thompson, MD, MPH, chief of the division of hematology, and Elias Schwartz, MD, Endowed Chair in Hematology at the Children's Hospital of Philadelphia.
23:06 | Dr. Thompson explains how sickle cell disease affects the body and its prevalence across the globe.
26:24 | What is the current treatment landscape for sickle cell disease?
30:03 | Victoria Gray shares her experience receiving a gene editing therapy for sickle cell disease.
33:26 | How do genetic therapies work for sickle cell disease?
37:26 | Clinical trials of gene therapy and gene editing approaches showed reduction in pain and improvements in other patient-reported outcomes in sickle cell patients.
46:32 | What are the risks and other factors that could affect gene therapy outcomes in sickle cell disease?
50:31 | What are the barriers to treatment access in the global sickle cell population?
52:00 | Closing remarks and introduction of Paul McCray, Jr., MD, professor and chief scientific officer in the department of pediatrics and division of pulmonary medicine at the Carver College of Medicine at the University of Iowa
53:50 | Dr. McCray introduces the overarching goal of genetic therapies for CF.
56:25 | Why does CF require a different approach than other genetic diseases, such as sickle cell?
1:00:49 | How different vectors are used to deliver genetic therapies to cells
1:04:12 | Gina Ruiz shares her CF story, including the physical and emotional challenges of not being eligible for CFTR modulators.
1:08:28 | Gene addition with a lentivirus can restore CFTR function in human airway cells in the lab.
1:11:30 | Several gene addition approaches are entering clinical trials in CF, with encouraging early results.
1:16:38 | Base editing, a new gene editing approach, could restore CFTR function for point mutations such as the R553X mutation.
1:20:05 | Super-exon insertion is a strategy that could be applied one time to repair most CFTR mutations.
1:23:44 | Closing remarks

Originally recorded November 2, 2023.