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ASGCT Podcast Network
02 - Kathy High, MD - Giants of Gene Therapy
Dec 20, 2022
Kathy High, MD, a pioneer in gene therapy and former president of Spark Therapeutics, shares her inspiring journey from working with hemophilia to advancing retinal disease treatments. Joined by Hans-Peter Kiem, MD, PhD, they delve into the challenges of developing gene therapies, including the complexities of AAV vectors and the hurdles of clinical trials for children. They also discuss recent innovations like synthetic AAVs, the importance of collaboration in biotech research, and offer wisdom for early-career hematologists pursuing their passions.
53:43
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Quick takeaways
- Dr. Kathy High's interdisciplinary background in chemistry and medicine has been essential in advancing gene therapy from research to clinical applications.
- Current innovations in gene therapy, such as synthetic AAV libraries and gene editing technologies, promise to enhance treatment efficacy and address key challenges.
Deep dives
Early Interests in Science and Medicine
Dr. Kathy Hye's early fascination with science was significantly influenced by her family's background, particularly her grandfather, a chemist. She developed an interest in chemistry at a young age, spurred by receiving a chemistry set from Santa Claus, which led her to a major in chemistry at Harvard. Initially uncertain about her career path, she contemplated both graduate school and medical school, ultimately deciding to pursue medicine after working in a research lab at Mass General that focused on metabolic disorders. This foundation in chemistry and her experiences in the lab solidified her commitment to a career where she could merge her scientific knowledge with medicine.
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