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Translational Medicine / 转化医学Gene Therapy Translation, Clinical Development

Katherine High

MD

🏢AskBio / formerly Children's Hospital of Philadelphia🌐USA

Former President, Therapeutics

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👥Biography 个人简介

Katherine High was a pioneer in translating gene therapy from laboratory concept to clinical reality, leading the first successful AAV gene therapy trials for hemophilia B that demonstrated sustained therapeutic protein expression from a single vector administration. Her clinical translation expertise has been applied to cancer gene therapy programs, particularly in manufacturing scalable AAV and lentiviral vectors meeting clinical-grade standards. She has navigated regulatory pathways for gene therapy approval and established safety monitoring frameworks applicable to cancer gene therapy. Her experience bridging gene therapy from rare disease to oncology applications has informed the design of cancer gene therapy clinical trials.

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🧪Research Fields 研究领域

gene therapy clinical translation
AAV gene therapy hemophilia
gene therapy regulatory pathway
gene therapy manufacturing scale-up
gene therapy safety monitoring

🎓Key Contributions 主要贡献

Representative Works 代表性著作

📄Data Sources 数据来源

Last updated: 2026-04-01 | All information from publicly available academic sources

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