ASGCT 13th Annual Meeting: Katherine High Selected as Outstanding Achievement Honoree


WASHINGTON – Katherine A High, MD, has been selected as the recipient of the Outstanding Achievement Award by the American Society of Gene & Cell Therapy.

Dr. High, who conducts her research at The Children’s Hospital of Philadelphia, was recognized for her extensive body of work in the gene therapy field. Her most notable achievements include advancements in treatments for hemophilia, a rare bleeding disorder, and Leber’s congenital amurosis, an inherited blindness.

Dr. High’s initial focus was on hemophilia and related bleeding disorders. She successfully isolated the gene responsible for blood clotting and has lead several studies in humans and dogs. While the animal trials produced long-term improvements in blood clotting, the effect in human patients was reliable but, to this point, short-lived.

“Dr. High has been a pioneer in the use of gene therapy to treat genetically inherited disorders. Her early work using dogs for testing of gene therapy for hemophilia and her subsequent patient applications of this process has moved the field forward in her discovery that animal models don't provide all the answers,” said John J. Rossi, PhD, ASGCT Advisory Council chairman. “Despite the failure of long-term success in the human hemophilia trial, Dr. High persisted to understand the cause of the disappointment – which she determined was an immune response to the vector.”

Dr. High applied those findings in gene therapy trials that have produced long-lasting sight restoration in children and adults with the heredity blindness Leber’s congenital amurosis. Ongoing trials are testing the hemophilia treatment with a different vector and in conjunction with immuno-suppressing agents to attempt to produce long-term correction in humans.

Well-constructed clinical trials of gene therapy have uncovered problems that were not predicted by studies in animals. Throughout her career, Dr. High has carefully analyzed these responses, laying the foundation for continued expansion of the applications of gene therapy in a safe and effective manner.

Katherine A. High, MD
Dr. High is presently William H. Bennett Professor of Pediatrics at the University of Pennsylvania School of Medicine, Investigator, Howard Hughes Medical Institute, and Director, Center for Cellular and Molecular Therapeutics at The Children’s Hospital of Philadelphia. Dr. High is a Past-President of ASGCT (2004-05).

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The Outstanding Achievement Award winner is selected by the ASGCT Advisory Council as an example of an ASGCT member who has achieved a pioneering research success, a specific high-impact accomplishment or a lifetime of significant scientific contributions to the fields of gene and cell therapy. The award recipient presents on her accomplishments during the Presidential Symposium at the ASGCT Annual Meeting.

The American Society of Gene & Cell Therapy (ASGCT) is a professional non-profit medical and scientific organization dedicated to the understanding, development and application of genetic and cellular therapies and the promotion of professional and public education in the field. For more information on ASGCT, visit its website,

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