Katherine A. High
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Katherine A. High is an American
doctor Doctor, Doctors, The Doctor or The Doctors may refer to: Titles and occupations * Physician, a medical practitioner * Doctor (title), an academic title for the holder of a doctoral-level degree ** Doctorate ** List of doctoral degrees awarded b ...
-scientist who is an
emeritus professor ''Emeritus/Emerita'' () is an honorary title granted to someone who retires from a position of distinction, most commonly an academic faculty position, but is allowed to continue using the previous title, as in "professor emeritus". In some c ...
at the
Perelman School of Medicine The Perelman School of Medicine (commonly known as Penn Med) is the medical school of the University of Pennsylvania, a private, Ivy League research university located in Philadelphia. Founded in 1765, the Perelman School of Medicine is the olde ...
at the
University of Pennsylvania The University of Pennsylvania (Penn or UPenn) is a Private university, private Ivy League research university in Philadelphia, Pennsylvania, United States. One of nine colonial colleges, it was chartered in 1755 through the efforts of f ...
. She was the co-founder, president, and chief scientific officer of
Spark Therapeutics Spark Therapeutics, Inc. is a developer of gene therapy treatments, which treat debilitating genetic diseases. It was founded in 2013 and has been a subsidiary of Hoffmann-La Roche since 2020. History The company was founded in 2013 by Katherin ...
and currently serves as President of Therapeutics at AskBio. She has worked in the area of
gene therapy Gene therapy is Health technology, medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells. The first attempt at modifying human DNA ...
, performing both basic research and clinical investigations. She has been recognized for her distinguished contributions to the field, having designed, sponsored, and conducted the first clinical trial of an
adeno-associated viral vector A viral vector is a modified virus designed to deliver genetic material into cells. This process can be performed inside an organism or in cell culture. Viral vectors have widespread applications in basic research, agriculture, and medicine. V ...
(AAV) gene therapy injected into the skeletal muscle (1999), the first trial of AAV gene therapy introduced into the liver (2001), and the first trial in the US of an AAV gene therapy injected into the subretinal space (2007).


Education

High received an A.B. in chemistry at Harvard University in 1972. She started her medical training at the
University of North Carolina The University of North Carolina is the Public university, public university system for the state of North Carolina. Overseeing the state's 16 public universities and the North Carolina School of Science and Mathematics, it is commonly referre ...
(UNC) School of Medicine, during which she took a leave of absence to work in a chemistry research lab at UNC. After returning and completing her medical degree in 1978, High continued with a residency in internal medicine. She then became a hematology fellow at
Yale University Yale University is a Private university, private Ivy League research university in New Haven, Connecticut, United States. Founded in 1701, Yale is the List of Colonial Colleges, third-oldest institution of higher education in the United Stat ...
under the supervision of Edward J Benz Jr, during which she worked on the molecular genetics of globin genes and oncogenes.


Career and research

High was a faculty member at the University of North Carolina - Chapel Hill for seven years, where she started her career by developing a canine model of the study of gene therapy for
hemophilia Haemophilia (British English), or hemophilia (American English) (), is a mostly inherited genetic disorder that impairs the body's ability to make blood clots, a process needed to stop bleeding. This results in people bleeding for a long ...
by cloning the canine
Factor IX Factor IX (), also known as Christmas factor, is one of the serine proteases involved in coagulation; it belongs to peptidase family S1. Deficiency of this protein causes haemophilia B. It was discovered in 1952 after a young boy named Stephen ...
gene. She also identified the mutations responsible for several
Factor VII Coagulation factor VII (, formerly known as proconvertin) is a protein involved in coagulation and, in humans, is encoded by gene ''F7''. It is an enzyme of the serine protease class. Once bound to tissue factor released from damaged tissues, ...
and
Factor X Coagulation factor X (), or Stuart factor, is an enzyme of the coagulation cascade, encoded in humans by ''F10'' gene. It is a serine endopeptidase (protease group S1, PA clan). Factor X is synthesized in the liver and requires vitamin K for i ...
-deficient blood clotting disorders. High moved to the University of Pennsylvania and
Children's Hospital of Philadelphia The Children's Hospital of Philadelphia, also known by its acronym CHOP, is a children's hospital in Philadelphia, Pennsylvania. Its primary campus is located in the University City, Philadelphia, University City neighborhood of West Philadelph ...
, where she began pioneering gene therapies for blood disorders. During her career at the University of Pennsylvania, High expanded her research into gene therapy solutions for hereditary blindness together with Dr. Jean Bennett. She was the director of the Center for Cellular and Molecular Therapeutics, and head of
hematology Hematology (American and British English spelling differences#ae and oe, spelled haematology in British English) is the branch of medicine concerned with the study of the cause, prognosis, treatment, and prevention of diseases related to bloo ...
research, at the Children's Hospital of Philadelphia and an investigator of the
Howard Hughes Medical Institute The Howard Hughes Medical Institute (HHMI) is an American non-profit medical research organization headquartered in Chevy Chase, Maryland with additional facilities in Ashburn, Virginia. It was founded in 1953 by Howard Hughes, an American busin ...
. High is an elected member of the National Academy of Medicine of the
American Academy of Arts and Sciences The American Academy of Arts and Sciences (The Academy) is one of the oldest learned societies in the United States. It was founded in 1780 during the American Revolution by John Adams, John Hancock, James Bowdoin, Andrew Oliver, and other ...
, and of the Faculty of Pharmaceutical Medicine of the Royal College of Physicians (London). Along with
Jean Bennett Jean Bennett is the F. M. Kirby Professor of Ophthalmology in the Perelman School of Medicine at the University of Pennsylvania. Her research focuses on gene therapy for retinal diseases. Her laboratory developed the first FDA approved gene the ...
, in 2018, she was awarded
Sanford Health Sanford Health is a nonprofit, integrated health care delivery system headquartered in Sioux Falls, South Dakota, with additional offices in Fargo and Bismarck, North Dakota, and Bemidji, Minnesota. History Sanford Health has its roots in ...
's $1 million Lorraine Cross Award for innovation in medicine and science. From 2014 to 2020, High served as the co-founder, President, Chief Scientific Officer/Head of R&D and a Member of the Board of Directors of Spark Therapeutics, a fully integrated, commercial gene therapy company in Philadelphia. While at Spark Therapeutics, Dr. High led the team that obtained the first FDA approval of an AAV therapeutic ( Voretigene neparvovec for the treatment of an inherited disorder causing blindness) in December 2017 and led the team that obtained Breakthrough Therapy designation and FDA approval for Fidanacogene elaparvovec to treat
Hemophilia B Haemophilia B, also spelled hemophilia B, is a blood clotting disorder causing easy bruising and bleeding due to an inherited mutation of the gene for factor IX, and resulting in a deficiency of factor IX. It is less common than factor VIII defic ...
and Breakthrough Therapy designation for Dirloctogene samoparvovec to treat
Hemophilia A Haemophilia A (or hemophilia A) is a blood clotting disorder caused by a genetic deficiency in clotting factor VIII, thereby resulting in significant susceptibility to bleeding, both internally and externally. This condition occurs almost exclu ...
. Spark Therapeutics was bought by Swiss pharma company
Roche F. Hoffmann-La Roche AG, commonly known as Roche (), is a Switzerland, Swiss multinational corporation, multinational holding healthcare company that operates worldwide under two divisions: Pharmaceuticals and Diagnostics. Its holding company, ...
in December 2019 for $4.3 billion. In February 2020, High stepped down from her position at the company. In January 2021, High joined Asklepios Biopharmaceutical, or AskBio, a clinical-stage adeno-associated virus (AAV) gene therapy company and wholly owned subsidiary of
Bayer AG Bayer AG (English: , commonly pronounced ; ) is a German multinational pharmaceutical and biotechnology company and is one of the largest pharmaceutical companies and biomedical companies in the world. Headquartered in Leverkusen, Bayer's ...
, as the company's new President of Therapeutics and as a board member for AskBio. In this role, Dr. High is responsible for driving the strategic direction and execution of the company's preclinical and clinical programs. In April 2021, she was elected a member of the U. S.
National Academy of Sciences The National Academy of Sciences (NAS) is a United States nonprofit, NGO, non-governmental organization. NAS is part of the National Academies of Sciences, Engineering, and Medicine, along with the National Academy of Engineering (NAE) and the ...
.


Personal life

She is married to George Steele, a nutritionist who was on the faculty of the University of Pennsylvania School of Medicine. She has three children, one of whom is actress
Sarah Steele Sarah Jane Steele (born September 16, 1988) is an American actress. She is known for her role as Marissa Gold on the CBS legal drama series ''The Good Wife'' (2011–2016) and its CBS All Access spinoff series ''The Good Fight'' (2017–2022). ...
.


References


External links


Penn Bio
{{DEFAULTSORT:High, Katherine A. Living people University of Pennsylvania faculty Harvard University alumni University of North Carolina School of Medicine alumni Fellows of the American Academy of Arts and Sciences Members of the National Academy of Medicine American hematologists Gene therapy Howard Hughes Medical Investigators Year of birth missing (living people) Perelman School of Medicine at the University of Pennsylvania faculty Women hematologists 20th-century American women physicians 20th-century American physicians 21st-century American women physicians 21st-century American physicians American women academics Members of the United States National Academy of Sciences