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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.

    Topics: Gene Editing, Plenary, Clinical Research
  • 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.

    Topics: CFTR, Plenary, Clinical Research
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