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Plenary
PL1- Genetic Therapies for All: Harnessing Cross-Disease Knowledge for Breakthroughs in Cystic Fibrosis
Thursday, November 2, 2023
4:30 PM – 6:00 PM MST
Location: North Ballroom A-D
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.
Light snacks and drinks will be provided outside the North Ballroom to enjoy before the Plenary.
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.