ASGCT and NORD

We've partnered with the National Organization for Rare Disorders to create a series of digital events designed to educate both patient and clinical audiences about the potential of gene therapy.

ASGCT's First-Ever CME Program

The Time Is Now: Understanding Gene Therapy, Applications, and Implications is a two-part CME series in partnership with NORD designed to explain the potential of gene‐based treatment approaches to change the disease course for patients with genetic conditions, discuss different types of current and emerging gene‐based therapies, and more!

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Past Events

Gene Therapy: Yesterday, Today and Tomorrow

ASGCT experts Cenk Sumen, Ph.D., and Phillip Tai, Ph.D., discuss the past, present, and future of gene therapy.

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The Science Behind Gene Therapy

Explore how gene therapies are developed, how evidence-based science guides gene therapy manufacturers, how vectors deliver gene therapies to cells, and what happens inside the body after gene therapy is delivered with speakers Sven Kili, M.D., and Tiffany Lucas, Ph.D.

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The FDA's Role in Gene Therapy

Discover regulatory pathways for gene therapies, how gene therapies are deemed safe, and more with speakers including Peter Marks, M.D., PhD, and Julie Tierney, JD

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Understanding the Gene Therapy Process and Aftercare

How patients are identified as candidates for gene therapy? Who may be on a gene therapy treatment team? How long do procedure takes? What can you expect during recovery and longer term follow up? Hear from ASGCT members Jerry Mendell, MD and Barry Byrne, MD, PhD.

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Life After Gene Therapy

Hear the stories from the sources themselves: Charles Hough was diagnosed with sickle cell disease when he was two years old and received gene therapy through a clinical trial at NIH in 2017. Nicole Almeida's son, Matteo, who received gene therapy when he was just 27 days old to treat SMA.

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2024

Breakthroughs in Muscular Dystrophy

November 19-20, 2024 | Chicago, IL

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