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Plenary II: Thursday, November 4, 2021
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.
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Plenary III: Friday, November 5, 2021
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.
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Plenary Session II: Thursday, October 22, 2020
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.
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Plenary III: Friday, October 23, 2020
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.
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Plenary Session I: Thursday, October 18, 2018
"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.
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Plenary Session II: Friday, October 19, 2018
"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.