Archives

Laying the Foundation for the Path to a Cure

Speakers: Brian R. Davis, Ph.D. and Maria P. Limberis, Ph.D.

To lay the foundation for the Path to a Cure, an ambitious initiative to accelerate treatments for the underlying cause of the disease and a cure for cystic fibrosis, we need to understand the cells to target and the challenges of delivering genetic-based therapies (such as gene editing and gene therapy).

In this plenary, Brian Davis, Ph.D. from the University of Texas Health Science Center at Houston, will focus on the importance of understanding the underlying cellular architecture and biology of the epithelial tissues (e.g., the lungs) that must be treated. Maria Limberis, Ph.D. from the University of Pennsylvania, will explore the complex physical properties of the lungs that make the surface of the airway an effective barrier against the viral and bacterial pathogens, but that also make the successful delivery of genetic-based therapies more difficult.

Their presentations will provide insight into the current status of promising state-of-the-are genetic-based therapies as well as put in context a common vocabulary and conceptual framework as we move forward with developing a cure for all.

"Emerging Technologies for CFTR Restoration in All People With CF"

Speaker: Marie Egan, M.D.

Genetic therapies, including RNA therapies, gene therapies, and gene editing, hold the key to a cure for cystic fibrosis and are moving toward the clinical realm at an unexpected pace. Moving from the promise of these technologies to the reality of effective therapies for the underlying cause of the disease for all people with CF and a cure for CF will require time, tenacity, and vision.

Marie Egan, MD, from the Yale School of Medicine, reviews the power of genetic therapies, outlines the obstacles and challenges that lie ahead, and discusses what success looks like as we move toward therapies that address the underlying defect for all people with CF and a cure for cystic fibrosis. 

"A Cure for All: Leaving No One Behind"

Speakers: Mitchell Drumm, Ph.D. and Eric Sorscher, M.D.

This session describes recent advances toward treatment of cystic fibrosis and the pipeline for new and emerging therapies.