Plenary 1: Challenges & Progress in the Pursuit of Genetic Therapies for Cystic Fibrosis
Presentation Date: Thursday, September 26, 2024
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 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.
Plenary 2: The Tipping Point: The Journey to Understand & Advance Research in CFRD
Presentation Date: Friday, September 27, 2024
Cystic fibrosis-related diabetes (CFRD) is an increasingly prevalent condition, affecting up to a quarter of adolescents and half of adults with CF, and often poses significant health challenges. The primary defect underlying CFRD is the pancreatic beta cell's inability to secrete sufficient insulin to maintain normal blood glucose levels. Despite recent advances in our understanding of the pancreatic processes that may contribute to beta cell dysfunction, we have much to learn about why CFRD develops and how to prevent it. Additionally, as the CF population ages and undergoes pulmonary and nutritional improvements — alongside rising obesity rates — the landscape of CFRD is evolving. Plenary 2 will focus on recent advances and exciting new directions in basic science and clinical research in the field of CFRD. Rebecca Hull-Meichle, PhD, will give us a view of the CF pancreas under a microscope, focusing on new insights into cellular mechanisms underlying CFRD and how these discoveries may one day be translated into potential therapies. Melissa Putman, MD, will take us through the changing landscape of CFRD in the post-modulator era, including emerging diabetes technologies, new treatment strategies, and redefining nutrition in an aging CF population.
Learning Objectives:
- Describe the complex cellular processes, contributing to the development of CFRD.
- Identify promising new research aimed at advancing our understanding of the CF pancreas.
- Describe opportunities for improving the screening, diagnosis, and treatment of CFRD using new diabetes technologies.
- Describe ongoing and future clinical studies seeking to expand treatment options, reduce burden, enhance access, and inform best practices in the post-modulator era.
Plenary 3: Reproductive Health in Cystic Fibrosis
Presentation Date: Saturday, September 28, 2024
As more people with cystic fibrosis are living longer, healthier lives, they increasingly face questions about their reproductive futures. This plenary will feature the experiences of people with CF and provide an overview of key research on reproductive health in CF and implications for CF care delivery. Molly Pam will highlight community engagement in this work and personal family-building decisions. Raksha Jain, MD, will review fertility and pregnancy among people with CF. Traci Kazmerski, MD will explore the interplay between CF and reproductive health decisions and implications for CF clinical care.
Learning Objectives:
- Discuss the changing dynamics of fertility and pregnancy for females with CF and potential implications for their infants in the modern era.
- Review male infertility and the journey through assisted reproductive technology.
- Summarize ongoing research on the interplay between CF and reproductive health and potential clinical interventions to improve care.
