Katherine A. High

Katherine Ann High, M.D., is President of Therapeutics at AskBio (Asklepios BioPharmaceutical, a wholly owned subsidiary of Bayer AG), a board member, and an accomplished hematologist with a longstanding interest in gene therapy for genetic diseases. She was elected a member of the U.S. National Academy of Sciences in 2021 and the American Academy of Arts and Sciences in 2011. Dr. High is internationally recognized as a pioneer in clinical translation of AAV gene therapy for hemophilia. After completing medical training in hematology at Yale, she joined Children's Hospital of Philadelphia (CHOP) in 1992 to study adeno-associated viral (AAV) vectors. From 2004 to 2014, she served as inaugural Director of CHOP's Center for Cellular and Molecular Therapeutics (CCMT), Professor at the University of Pennsylvania's Perelman School of Medicine, and Howard Hughes Medical Institute Investigator. High's pioneering bench-to-bedside studies of gene therapy for hemophilia led to landmark clinical trials that systematically characterized the human immune response to AAV vectors in various target tissues. Her 2011 and 2014 New England Journal of Medicine publications reported the first clinical trials demonstrating safety and long-term efficacy of AAV8-mediated factor IX gene transfer in hemophilia B patients, providing critical proof-of-concept that directly catalyzed development of FDA-approved hemophilia gene therapies including Hemgenix (2022, first FDA-approved gene therapy for hemophilia). In 2014, High left CHOP to co-found Spark Therapeutics, serving as President and Chief Scientific Officer until 2020. Under her leadership, Spark developed Luxturna (voretigene neparvovec)—approved by FDA in 2017 as the first gene therapy for inherited retinal disease—and hemophilia A gene therapy fidanacogene elaparvovec (licensed to Pfizer). Swiss pharma company Roche acquired Spark for $4.3 billion in 2019, validating the commercial potential of AAV therapeutics. Since joining AskBio in January 2021 as President of Therapeutics, High continues advancing AAV gene therapy clinical development. She received the 2025 Bengt Samuelsson Award from Lund University and CHOP's Gold Medal for her pioneering contributions to gene therapy.

Katherine Ann High医学博士，是AskBio（Asklepios BioPharmaceutical，拜耳全资子公司）治疗总裁、董事会成员，是一位成就卓著的血液学家，长期致力于遗传疾病基因治疗。她于2021年当选为美国国家科学院院士，2011年当选为美国艺术与科学院院士。 High博士国际公认为血友病AAV基因治疗临床转化的先驱。在耶鲁完成血液学医学培训后，她于1992年加入费城儿童医院(CHOP)研究腺相关病毒(AAV)载体。从2004年到2014年，她担任CHOP细胞与分子治疗中心(CCMT)首任主任、宾夕法尼亚大学佩雷尔曼医学院教授和霍华德·休斯医学研究所研究员。 High的血友病基因治疗先驱性从实验室到病床研究导致了里程碑式临床试验，系统地表征了人类对各种靶组织中AAV载体的免疫反应。她2011年和2014年发表在《新英格兰医学杂志》上的论文报告了首批临床试验，证明AAV8介导的凝血因子IX基因转移在血友病B患者中的安全性和长期有效性，提供了关键概念验证，直接催化了FDA批准的血友病基因治疗的发展，包括Hemgenix（2022年，首个FDA批准的血友病基因治疗）。 2014年，High离开CHOP共同创建Spark Therapeutics，担任总裁兼首席科学官直至2020年。在她的领导下，Spark开发了Luxturna (voretigene neparvovec)——2017年FDA批准为首个遗传性视网膜疾病基因治疗——以及血友病A基因治疗fidanacogene elaparvovec（授权给辉瑞）。瑞士制药公司罗氏于2019年以43亿美元收购Spark，验证了AAV治疗的商业潜力。 自2021年1月加入AskBio担任治疗总裁以来，High继续推进AAV基因治疗临床开发。她获得了2025年隆德大学Bengt Samuelsson奖和CHOP金质奖章，表彰她对基因治疗的开创性贡献。