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| Dr. Katherine High | |
|---|---|
| Name | Katherine High |
| Birth date | 1959 |
| Nationality | American |
| Fields | Hematology, Gene Therapy, Molecular Biology |
| Institutions | University of Pennsylvania, Children's Hospital of Philadelphia, Spark Therapeutics |
| Awards | Lasker~DeBakey Clinical Medical Research Award, National Academy of Medicine |
Dr. Katherine High Katherine Anne High is an American physician-scientist and hematologist noted for pioneering work in gene therapy for inherited bleeding disorders. She led translational research bridging laboratory discoveries and clinical application, directed academic programs and co-founded a biotechnology company that advanced adeno-associated virus vectors into licensed therapies. Her career spans academic medicine, translational research, industry leadership, and policy-influencing advisory roles.
High was born in the United States and trained in medicine and research at major institutions associated with leaders in biomedical science. She earned her MD at Columbia University Vagelos College of Physicians and Surgeons where she encountered influential figures linked to Columbia University, NewYork-Presbyterian Hospital, Harvard Medical School-affiliated networks, and peers from Johns Hopkins University and Yale University. Her postgraduate training included residency and fellowship experiences connected to clinical centers like Massachusetts General Hospital and research groups from National Institutes of Health laboratories. Mentors and collaborators from institutions such as Stanford University, University of California, San Francisco, Washington University in St. Louis, and University of Michigan helped shape her translational focus.
High completed specialty training in hematology at leading clinical and research centers, working alongside investigators affiliated with Dana-Farber Cancer Institute, Fred Hutchinson Cancer Research Center, Mayo Clinic, and Cleveland Clinic. Her early research investigated coagulation factors and molecular pathogenesis of bleeding disorders, interacting with contemporaries from Imperial College London, University College London, Karolinska Institutet, and Institut Pasteur. She held faculty appointments at the University of Pennsylvania and leadership roles at Children's Hospital of Philadelphia, collaborating with teams linked to Broad Institute, Perelman School of Medicine, Rockefeller University, and Cold Spring Harbor Laboratory. Her laboratory published alongside authors from Nature Medicine, The New England Journal of Medicine, Science Translational Medicine, and Blood contributors drawn from University of Cambridge, Oxford University, McGill University, and University of Toronto.
High was instrumental in developing adeno-associated virus (AAV)–based gene transfer for hemophilia, coordinating translational pathways involving vector design and clinical protocols with investigators from University of Florida, University of North Carolina at Chapel Hill, University of Pennsylvania Perelman School of Medicine, and regulatory scientists at the Food and Drug Administration. Her work focused on factor IX and factor VIII gene delivery, interacting with researchers at Genentech, Roche, Novartis, and academic groups at Imperial College London and Karolinska Institutet. She collaborated with clinical trial leaders connected to Mayo Clinic, Cleveland Clinic, Massachusetts General Hospital, and Johns Hopkins University to demonstrate durable expression and clinical benefit in adults with hemophilia, contributing evidence cited by panels at National Institutes of Health workshops and advisory committees of European Medicines Agency. Her translational programs paralleled efforts by teams at University of Oxford, Sanofi, Pfizer, Bayer, and academic consortia including Gene Therapy Program networks, and intersected with ethical and safety discussions led by scholars at Harvard Kennedy School, Georgetown University, and University of California, Berkeley.
High co-founded and served as chief scientific officer of a biotechnology company that advanced an AAV-based genetic medicine from bench to market, engaging with investors and partners from Glossary Capital, ARCH Venture Partners, Sequoia Capital, and SV Health Investors as well as strategic collaborators at Spark Therapeutics, UniQure, Regenxbio, Bluebird Bio, and CRISPR Therapeutics. She guided interactions with regulatory and reimbursement stakeholders including the Food and Drug Administration, European Medicines Agency, and payers influenced by reports from Institute for Clinical and Economic Review. Her leadership integrated translational teams across Children's Hospital of Philadelphia, University of Pennsylvania Health System, and corporate R&D groups at Roche and Novartis, while participating in advisory boards for Bill & Melinda Gates Foundation, Wellcome Trust, Howard Hughes Medical Institute, and industry consortia with BIO and PhRMA.
High's contributions earned recognition from major organizations and academies. She is an elected member of the National Academy of Medicine and has received awards such as the Lasker~DeBakey Clinical Medical Research Award, honors from the American Society of Hematology, fellowships from the American Association for the Advancement of Science, and distinctions associated with the Gairdner Foundation and Helen Hay Whitney Foundation. Professional societies including American Board of Internal Medicine, Association of American Physicians, American College of Physicians, and International Society on Thrombosis and Haemostasis have acknowledged her leadership. She has delivered named lectures at institutions like Harvard Medical School, Yale School of Medicine, Johns Hopkins University School of Medicine, and international venues including European Hematology Association congresses.
High's personal life has been private; she balanced clinical responsibilities, laboratory mentorship, and executive duties while influencing trainees who joined academic centers such as University of Pennsylvania, University of Michigan, Stanford University School of Medicine, and industry teams at Spark Therapeutics and Bluebird Bio. Her legacy includes training clinician-scientists who now hold appointments at Children's National Hospital, UCSF Benioff Children's Hospitals, Boston Children's Hospital, and shaping policy dialogues at the National Institutes of Health and global forums like the World Health Organization. Her work continues to inform gene therapy programs at academic, philanthropic, and commercial institutions worldwide.
Category:American hematologists Category:Women in biotechnology Category:Gene therapy researchers