Katherine A. High is an American doctor-scientist who is an emeritus professor at the Perelman School of Medicine at the University of Pennsylvania. She was the co-founder, president, and chief scientific officer of Spark Therapeutics and currently serves as President of Therapeutics at AskBio.[1] She has worked in the area of gene therapy, 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 (AAV) gene therapy injected into the skeletal muscle (1999),[2] the first trial of AAV gene therapy introduced into the liver (2001),[3] and the first trial in the US of an AAV gene therapy injected into the subretinal space (2007).[4]
Education
High received an A.B. in chemistry at Harvard University in 1972.[5][6] She started her medical training at the University of North Carolina (UNC) School of Medicine, during which she took a leave of absence to work in a chemistry research lab at UNC.[7] After returning and completing her medical degree in 1978, High continued with a residency in internal medicine.[8] She then became a hematology fellow at Yale University under the supervision of Edward J Benz Jr, during which she worked on the molecular genetics of globin genes and oncogenes.[9][8]
Career and research
High was a faculty member at the University of North Carolina - Chapel Hill for seven years,[10][8] where she started her career by developing a canine model of the study of gene therapy for hemophilia by cloning the canine Factor IX gene.[11] She also identified the mutations responsible for several Factor VII and Factor X-deficient blood clotting disorders.[12] High moved to the University of Pennsylvania and Children's Hospital of Philadelphia, where she began pioneering gene therapies for blood disorders.[13] During her career at the University of Pennsylvania, High expanded her research into gene therapy solutions for hereditary blindness together with Dr. Jean Bennett.[14]
She was the director of the Center for Cellular and Molecular Therapeutics, and as of 2001[update] head of hematology research,[1] at the Children's Hospital of Philadelphia and an investigator of the Howard Hughes Medical Institute.[15][16] High is an elected member of the National Academy of Medicine of the American Academy of Arts and Sciences, and of the Faculty of Pharmaceutical Medicine of the Royal College of Physicians (London).
Along with Jean Bennett, in 2018, she was awarded Sanford Health'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 and Breakthrough Therapy designation for Dirloctogene samoparvovec to treat Hemophilia A.
Spark Therapeutics was bought by Swiss pharma company Roche in December 2019 for $4.3 billion. In February 2020, High stepped down from her position at the company.[17]
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, 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.
She is married to George Steele, a nutritionist who was on the faculty of the University of Pennsylvania School of Medicine.[19][20] She has three children, one of whom is actress Sarah Steele.[21][22]
^Kay MA, Manno CS, Ragni MV, Larson PJ, Couto LB, McClelland A, Glader B, Chew AJ, Tai SJ, Herzog RW, Arruda V, Johnson F, Scallan C, Skarsgard E, Flake AW, High KA (2000). "Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector". Nature Genetics. 24 (3): 257–261. doi:10.1038/73464. PMID10700178. S2CID12650196.
^Manno CS, Arruda VR, Pierce GF, Glader B, Ragni M, Rasko JJ, Ozelo MC, Hoots K, Blatt, P, Konkle B, Dake M, Kaye R, Razavi M, Zajko A, Zehnder J, Nakai H, Chew A, Leonard D, Wright JF, Lessard RR, Sommer JM, Tigges M, Sabatino D, Luk A, Jiang H, Mingozzi F, Couto L, Ertl HC, High KA, Kay MA (2006). "Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response". Nature Medicine. 12 (3): 342–347. doi:10.1038/nm1358. PMID16474400. S2CID14664182.
^Maguire AM, Simonelli F, Pierce EA, Pugh EN Jr, Mingozzi F, Bennicelli J, Banfi S, Marshall KA, Testa F, Surace EM, Rossi S, Lyubarsky A, Arruda VR, Konkle B, Stone E, Sun J, Jacobs J, Dell'osso L, Hertle R, Ma JX, Redmond TM, Zhu X, Hauck B, Zelenaia O, Shindler KS, Maguire MG, Wright JF, Volpe NJ, McDonnell JW, Auricchio A, High KA, Bennett J (April 28, 2008). "Safety and efficacy of gene transfer for Leber's Congenital Amaurosis". New England Journal of Medicine. 358 (21): 2240–2248. doi:10.1056/NEJMoa0802315. PMC2829748. PMID18441370.
^Herzog, R. W.; Yang, E. Y.; Couto, L. B.; Hagstrom, J. N.; Elwell, D.; Fields, P. A.; Burton, M.; Bellinger, D. A.; Read, M. S.; Brinkhous, K. M.; Podsakoff, G. M. (January 1999). "Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector". Nature Medicine. 5 (1): 56–63. doi:10.1038/4743. ISSN1078-8956. PMID9883840. S2CID21832836.
^"News from the National Academy of Sciences". 26 April 2021. Retrieved 2 July 2021. Newly elected members and their affiliations at the time of election are: ... High, Katherine A.; president, Therapeutics, AskBio, Research Triangle Park, N.C.
