

02 - Kathy High, MD - Giants of Gene Therapy
8 snips 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.
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Early Influences
- Kathy High's grandfather, a chemist, sparked her early interest in science.
- This led her to major in chemistry at Harvard and later pursue medical school.
Path to Medicine
- Despite initial doubts about medicine, Kathy High persevered, even taking a year off to work in a chemistry lab.
- A mentor's advice and a renewed interest in medicine during her clinical rotations led her to complete medical school.
Hematology and Early Gene Therapy
- Kathy High chose hematology because it aligned with her chemistry background and allowed her to study hemophilia.
- Her research focused on identifying hemophilia-causing mutations, paving the way for gene therapy research.