Archives

Working Together to Pave the Path to a Cure

Speakers: Martin Mense, PhD, Hillary Valley, PhD, and John Mahoney, PhD

The success of the CF Foundation's Path to a Cure relies on industry and academia having the tools and resources necessary to test and develop promising scientific approaches and technologies, including those for gene editing, gene therapy (replacement) and genetic therapy delivery. The CF Foundation Therapeutics (CFFT) Lab plays a critical role in this process.

During Plenary 2 of NACFC 2021, Martin Mense Ph.D., Hillary Valley, Ph.D., and Jed Mahoney, Ph.D., from the CFFT Lab will outline the current challenges of developing cutting-edge therapies and explain what steps the lab and others are taking to remove these obstacles and accelerate progress towards new treatments for the underlying cause of the disease and, one day, a cure.

Expanding the Horizon of Therapies for the Underlying Cause of CF

Speaker: Manu Jain, MD, MSc

Over the last decade, highly effective CFTR modulators (HEM) have transformed the health and prospects for a large majority of people with CF. Nevertheless, there remains a substantial need to bring effective CFTR therapies to those with CF who lack treatment options for the underlying cause of their disease as well as those with sup-optimal responses or tolerability issues to approved drugs.

During plenary 3 of NACFC 2021, Manu Jain MD, MSc, will highlight recent data on the real world-impact of HEM as well as data supporting the use of CFTR modulators in new CF communities. He will also discuss emerging data on the pre-clinical and clinical development of genetic therapies that rescue, restore and/or replace CFTR as well as alternative strategies to target the underlying ion transport defects of the disease.

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.

Defining the New CF in the Era of Highly Effective Modulators

Speaker: Jennifer L. Taylor-Cousar, M.D., MSCS, ATS

In this session, the latest research into highly effective CFTR modulators will be highlighted, providing and in-depth overview of the CFTR modulator research landscape one-year post-approval of Trikafta. The speaker will describe the ongoing and upcoming studies looking at the holistic impact of highly effective CFTR modulation on the lives of people with CF. Finally, there will be a review of on-going and upcoming studies for the development of new modulators as well as the potential impact of modulators on complications.

Advancing GI Frontier for People with CF

Speaker: Steven D. Freedman, M.D., Ph.D.

In this session, Dr. Freedman walks us through the gastrointestinal aspects of CF, focusing on intestinal, liver, and pancreatic disease and the exciting new directions in GI as part of specialized CF care. Research initiatives and advances in developing a GI workforce through the CF Foundation's DIGEST program will be highlighted.

"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.