During this plenary, the speakers will explore different approaches to overcoming the biological obstacles and unique clinical trial challenges for developing cystic fibrosis genetic therapies. Daniel Siegwart, PhD, from the University of Texas Southwestern Medical Center, will discuss how scientists are leveraging emerging research to address the significant biological hurdles, such as overcoming physical barriers like mucus, targeting the correct types of cells, and strategies for delivery of genetic medicines to the lungs. Joe Pilewski, MD, from the University of Pittsburgh, will highlight how new research advances and approaches in translational science are crucial for developing effective genetic therapies for CF, and how taking innovative approaches to clinical trials and community engagement are essential to success.
Learning Objectives:
Identify biological challenges of developing a genetic therapy for cystic fibrosis.
Identify innovations in drug delivery technologies & and how they can support a variety of genetic therapies.
Describe how genetic therapies have the potential to restore CFTR function to the lungs, regardless of the underlying CFTR mutation.
Express how clinical trials for genetic therapies will require careful clinical trial design and increased community engagement.
