This article was accepted into the corpus but its outbound wikilinks were never NER-processed — typical at the deepest BFS hop or when the run's entity cap was reached. No expansion funnel to show.
| Katherine High | |
|---|---|
| Name | Katherine High |
| Birth date | 1958 |
| Nationality | American |
| Fields | Hematology, Gene therapy, Molecular biology |
| Alma mater | Duke University School of Medicine, Harvard Medical School |
| Workplaces | Children's Hospital of Philadelphia, Spark Therapeutics, University of Pennsylvania, Columbia University |
| Known for | Development of gene therapy for hemophilia, AAV vector clinical translation |
Katherine High is an American physician-scientist and hematologist noted for pioneering clinical translation of gene therapy for inherited bleeding disorders. She led research integrating molecular genetics, adeno-associated virus vectors, and translational medicine to treat hemophilia A, hemophilia B, and other monogenic disorders, and co-founded biotechnology firms to move therapies from academic laboratories to regulatory approval. Her career spans leadership roles at major research hospitals, biotech startups, and academic institutions, influencing clinical practice, regulatory pathways, and biotechnology entrepreneurship.
High was born in the United States and trained in medicine and molecular biology during an era shaped by advances at institutions such as Duke University School of Medicine, Harvard Medical School, and research centers like the National Institutes of Health. She completed medical education and residency training in settings affiliated with University of Pennsylvania, where clinical hematology programs intersected with research on coagulation factors such as factor VIII and factor IX. Her formative years overlapped with landmark discoveries in gene transfer, including foundational work on retrovirus and adeno-associated virus vectors and seminal gene therapy trials at centers including Children's Hospital of Philadelphia and Columbia University Irving Medical Center.
High's career combined roles in academic medicine, translational research, and biotech leadership. She held faculty and leadership positions at institutions such as Children's Hospital of Philadelphia, the University of Pennsylvania, and Columbia University, collaborating with investigators from laboratories at Massachusetts Institute of Technology, Stanford University School of Medicine, and Harvard Medical School. Her laboratory focused on molecular characterization of bleeding disorders like von Willebrand disease and genetic therapies targeting factor IX mutations. She collaborated with regulatory bodies including the U.S. Food and Drug Administration and patient organizations such as the National Hemophilia Foundation to design clinical trials and outcome measures. Transitioning into industry, she co-founded and served as chief scientific officer of biotechnology companies influenced by investment from firms like Perceptive Advisors and Novo Holdings and partnerships with pharmaceutical companies including Roche and Pfizer.
High's major contributions center on development and clinical translation of adeno-associated virus (AAV)-mediated gene transfer for hemophilia. She led preclinical and clinical programs demonstrating durable expression of therapeutic levels of clotting factors following systemic AAV delivery, building on vector engineering advances from labs such as James M. Wilson's group at University of Pennsylvania and capsid evolution strategies pioneered at Children's Hospital of Philadelphia. Her work contributed to pivotal clinical trials using AAV serotypes like AAV8 and AAV variants to target hepatocytes, informing dosing, immunomodulation strategies involving agents such as prednisone and protocols adopted by regulatory agencies including the European Medicines Agency. These programs culminated in approval pathways for gene therapies addressing hemophilia B and informed investigational approaches for hemophilia A, Leber congenital amaurosis, and other monogenic diseases treated with gene replacement or gene editing platforms like CRISPR-Cas9. Her translational efforts integrated manufacturing scale-up practices from contract development and manufacturing organizations associated with Good Manufacturing Practice frameworks and collaborations with translational networks including the Clinical and Translational Science Awards consortium.
High's recognitions reflect impact across medicine, genetics, and biotechnology. She received honors from professional societies such as the American Society of Hematology and the American Academy of Arts and Sciences, and awards acknowledging translational innovation from organizations like the National Academy of Medicine and biotechnology associations connected to BIO conferences. She has been elected to scientific bodies and advisory boards at institutions including NIH panels, invited to deliver lectures at meetings such as the International Society on Thrombosis and Haemostasis and featured in profiles by outlets focused on biomedical entrepreneurship and regulatory innovation.
High has authored and co-authored numerous peer-reviewed articles in journals such as The New England Journal of Medicine, Nature Medicine, Blood, Science Translational Medicine, and Nature Biotechnology describing AAV-mediated gene transfer trials, vector immunology, and long-term outcomes for clotting factor expression. Her publications often list collaborators from institutions including Children's Hospital of Philadelphia, University of Pennsylvania Perelman School of Medicine, Massachusetts General Hospital, and Stanford University. She is named on patents related to AAV vector design, promoter elements for liver-directed expression, and manufacturing processes, filed with agencies such as the United States Patent and Trademark Office and licensed to companies originating from academic spinouts.
Outside the laboratory, High has engaged with patient advocacy groups including the World Federation of Hemophilia and National Hemophilia Foundation to inform trial design, access, and ethics in gene therapy. She has participated in advisory roles for philanthropic funders like the Gates Foundation and foundations supporting rare disease research, and has mentored trainees affiliated with programs at Howard Hughes Medical Institute-supported institutions and international collaborations across Europe and Asia. Her public engagement has intersected with policy discussions involving regulators such as the U.S. Food and Drug Administration and professional guideline committees at societies like the International Society on Thrombosis and Haemostasis.
Category:American hematologists Category:Women physicians Category:Gene therapy researchers