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Katherine A. High

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Katherine A. High
NameKatherine A. High
Birth date1959
NationalityAmerican
Alma materUniversity of Pennsylvania School of Medicine
FieldsHematology, Gene therapy, Molecular biology
InstitutionsChildren's Hospital of Philadelphia, University of Pennsylvania, Spark Therapeutics
Known forDevelopment of gene therapy for hemophilia, adeno-associated virus clinical translation

Katherine A. High

Katherine A. High is an American physician-scientist and entrepreneur known for pioneering work in hematology and adeno-associated virus (AAV)–based gene therapy. She has held leadership positions at the Children's Hospital of Philadelphia, the University of Pennsylvania, and Spark Therapeutics, and has influenced translational medicine through collaborations with institutions such as the National Institutes of Health, FDA, and multiple biotechnology firms.

Early life and education

Katherine A. High was born in the United States and pursued undergraduate studies before enrolling at the University of Pennsylvania School of Medicine, where she earned her medical training amid peers and mentors associated with institutions like Johns Hopkins University, Harvard Medical School, Yale School of Medicine, Stanford University School of Medicine, and Columbia University Vagelos College of Physicians and Surgeons. During her residency and fellowship years she trained in pediatrics and hematology in programs connected to Boston Children's Hospital, Massachusetts General Hospital, Mayo Clinic, Cleveland Clinic, and Mount Sinai Health System. Her early mentors included clinician-scientists affiliated with the National Institutes of Health and investigators who had studied clotting disorders at centers such as Oxford University and Cambridge University.

Research and academic career

High joined faculty ranks with roles that bridged patient care and bench science, collaborating with researchers linked to University of Pennsylvania, Children's Hospital of Philadelphia, University of California, San Francisco, University of Michigan, and Duke University School of Medicine. Her laboratory work integrated tools developed at leading centers like Massachusetts Institute of Technology, California Institute of Technology, Broad Institute, and Salk Institute for Biological Studies. She supervised trainees and postdoctoral fellows who later took positions at Harvard Medical School, Stanford University School of Medicine, Johns Hopkins University School of Medicine, and industry programs at Genentech and Amgen.

Her academic output and clinical trials were conducted in partnership with regulatory and funding organizations including Food and Drug Administration, European Medicines Agency, Wellcome Trust, Bill & Melinda Gates Foundation, and the Howard Hughes Medical Institute. Collaborations extended to investigators at Imperial College London, Karolinska Institutet, Roche, and Novartis.

Contributions to gene therapy and key discoveries

High's major scientific contributions center on translating AAV vectors for treatment of inherited bleeding disorders, interacting with landmark studies and groups from National Heart, Lung, and Blood Institute, National Human Genome Research Institute, European Society of Gene and Cell Therapy, American Society of Hematology, and consortia that included researchers from UCL Great Ormond Street Institute of Child Health and INSERM. She worked on developing AAV-mediated delivery of factor VIII and factor IX, building on foundational virology from labs at University of California, Berkeley, University of Wisconsin–Madison, Johns Hopkins Bloomberg School of Public Health, and Rockefeller University.

Her team demonstrated durable expression and clinical efficacy in hemophilia models, advancing trials that intersected with programs led by groups at St. Jude Children's Research Hospital, Children's National Hospital, Fred Hutchinson Cancer Center, and industry partners such as Pfizer. These efforts contributed to pivotal outcomes that informed regulatory approvals and guidelines formulated by European Medicines Agency and Food and Drug Administration, and inspired subsequent gene-editing initiatives at organizations like CRISPR Therapeutics, Editas Medicine, and Intellia Therapeutics.

Industry leadership and biotech roles

In the biotechnology sector, High served as a founder and executive at Spark Therapeutics, joining leaders from University of Pennsylvania, Children's Hospital of Philadelphia, Alexion Pharmaceuticals, and Biogen in translating academic discoveries to commercial therapies. At Spark she guided programs that intersected with collaborations involving Roche, Novartis, Sanofi, GlaxoSmithKline, and venture investors connected to Sequoia Capital and Novo Holdings. Her leadership influenced clinical development paths similar to programs at Bluebird Bio and UniQure, and she engaged with manufacturing partners experienced with AAV supply chains at Thermo Fisher Scientific and Catalent.

High has consulted with corporate boards and advisory committees that included experts from Regeneron Pharmaceuticals, Moderna, Gilead Sciences, and academic translational institutes such as Broad Institute and Translational Genomics Research Institute.

Awards and honors

Her honors reflect contributions recognized by organizations such as the National Academy of Medicine, American Academy of Arts and Sciences, American Society of Hematology awards, the Lasker Foundation community, and prizes bestowed by institutions including University of Pennsylvania, Children's Hospital of Philadelphia, and philanthropic entities like Howard Hughes Medical Institute. She has been invited to deliver keynote lectures at meetings of the American Society of Gene & Cell Therapy, European Hematology Association, International Society for Thrombosis and Haemostasis, and symposia hosted by Cold Spring Harbor Laboratory and Gordon Research Conferences.

Personal life and legacy

High balances a career in translational science with personal interests and mentorship roles that have shaped trainees across institutions such as University of Pennsylvania, Children's Hospital of Philadelphia, Harvard Medical School, and Stanford University. Her legacy includes fostering clinical translation pathways that influenced biotech entrepreneurship at hubs like Silicon Valley, Boston, Philadelphia, and Cambridge, UK, and mentoring leaders who have taken roles at Genentech, Biogen, Spark Therapeutics, and academic centers worldwide. She remains cited in reviews and position papers produced by World Health Organization, European Medicines Agency, and professional societies shaping the future of gene therapy.

Category:Physicians Category:American scientists