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Watch Webinars from ASGCT and NORD

ASGCT and the National Organization for Rare Disorders (NORD) have partnered to produce a series of five patient education webinars.

Gene Therapy: Yesterday, Today and Tomorrow

In the first webinar, Cenk Sumen, Ph.D., chief technology officer of Stemson Therapeutics and adjunct professor at NYU's Tandon School of Engineering, and Phillip Tai, Ph.D., chair of ASGCT's  Patient Outreach Committee and a research instructor at the University of Massachusetts Medical School, discussed the past, present, and future of gene therapy.

The Science Behind Gene Therapy

The second webinar explored 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. The speakers were Sven Kili, M.D., a regenerative medicine consultant, and Tiffany Lucas, Ph.D., an FDA gene therapy product reviewer in the Center for Biologics Evaluation and Research (CBER).

The FDA's Role in Gene Therapy

The third webinar explored regulatory pathways for gene therapies, how gene therapies are deemed safe, and more. Speakers included Peter Marks, M.D., Ph.D., director of the FDA CBER; and Julie Tierney, J.D., senior policy advisory for strategic planning and legislation at CBER.

Understanding the Gene Therapy Process and Aftercare

The fourth webinar covered how patients are identified as candidates for gene therapy, who may be on a gene therapy treatment team, how long the procedure takes, and what to expect during recovery and longer term follow up. Participants heard from ASGCT Members Jerry Mendell, M.D., principal investigator in the Center for Gene Therapy at The Research Institute of Nationwide Children's Hospital; and Barry Byrne, M.D., Ph.D., director of the Powell Gene Therapy Center at the University of Florida. 

Life After Gene Therapy

During the fifth and final webinar in the series, participants heard from Charles Hough, who was diagnosed with sickle cell disease when he was two years old, and Nicole Almeida, who found out her son had SMA when she was five months pregnant. Charles received gene therapy through a clinical trial at NIH in 2017. Nicole's son, Matteo, received gene therapy when he was just 27 days old. Watch the webinar to learn more about Charles and Matteo's stories.

Last Updated: 02/10/2023

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