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.

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.

"Improving Outcomes of Infections in the Age of CFTR Modulators"

Speaker: Lisa Saiman, M.D., M.P.H.

In this session, Dr. Lisa Saiman will address the challenges of infections in people with CF and discuss why infections remain a major focus of the CF community, even in the era of effective CFTR modulators. She will provide an overview of CF infections, describe ongoing studies to increase our understanding of CF microorganisms and their optimal treatment, and discuss current strategies to develop new anti-infective agents to treat even the most difficult infections.

"Anti-inflammatories & Mucociliary Clearance Therapies in the Age of CFTR Modulators"

Speaker: Felix Ratjen, M.D., Ph.D.

This plenary will review the effect of CFTR dysfunction and CFTR restoration on both inflammation and mucociliary clearance in individuals with CF. Current and future therapeutic strategies to address inflammation and mucociliary clearance will be discussed, as well as how these strategies have potential to benefit both those patients using CFTR modulators and those not eligible for modulators.