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25min chapter
02 - Kathy High, MD - Giants of Gene Therapy
ASGCT Podcast Network
Challenges and Triumphs in Gene Therapy Development
This chapter explores the journey of gene therapy from animal models to human clinical trials, emphasizing the roles of AAV vectors and the hurdles faced during research. It highlights collaborative efforts between scientists and business leaders, the regulatory challenges in including children in trials, and the intricacies of transitioning from academia to biotech. Ultimately, the chapter reflects on the importance of manufacturing standards and overcoming past setbacks to ensure the advancement of gene therapy.
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