(WASHINGTON, August 1, 2013) - The American Society of Hematology (ASH) will honor Katherine A. High, MD, of the Howard Hughes Medical Institute and The Children's Hospital of Philadelphia with the 2013 E. Donnall Thomas Lecture and Prize for her unparalleled work to identify the molecular basis of hemophilia and to develop novel genetic therapies to treat the disorder. Named for the late Nobel Prize laureate and past Society president E. Donnall Thomas, MD, this lectureship and prize recognizes pioneering research achievements in hematology that have helped move the field forward.
Dr. High will present her lecture, "Sailing to Ithaca: Gene Therapy's Odyssey from Investigational Agent to Therapeutic Product," at 9:00 a.m. on Monday, December 9, at the 55th ASH Annual Meeting and Exposition in New Orleans. Her lecture will explore the use of gene therapies for the treatment of inherited disorders, discussing the therapeutic successes of laboratory and small-scale clinical studies conducted over the last two decades as well as the obstacles that remain to bring this seemingly straightforward yet extremely nuanced concept to successful clinical application. During her lecture Dr. High will also discuss the sustained clinical improvements yielded in the setting of inherited retinal degenerative diseases and of hemophilia B by the use of in vivo gene transfer with adeno-associated virus (AAV) vectors. She will reflect on how a rigorous understanding of the interaction of AAV vectors with the human immune response, and other homeostatic mechanisms, has allowed investigators to harness the power of gene identification and gene delivery vectors to provide long-lasting therapeutic outcomes for previously incurable diseases.
As a leading expert in gene therapy of hemophilia, Dr. High has dedicated her career to bringing this novel and complex concept to fruition. Her work has ranged from basic biochemical investigations of clotting factors, virology of the AAV viruses as relevant to vector design, dissection of the human immune response to vector and transgene proteins, development of predictive large animal models, and iterative careful and highly instructive clinical trials. Her more than 15 years of investigation regarding the feasibility of gene therapy for hemophilia has culminated in successful clinical studies utilizing AAV vector-mediated gene transfer for the treatment of hemophilia B.
Dr. High is a Howard Hughes Medical Institute Investigator, the William H. Bennett Professor of Pediatrics at the University of Pennsylvania School of Medicine, and an attending physician at the Children's Hospital of Philadelphia. She received her medical degree from the University of North Carolina School of Medicine and completed her Hematology fellowship at Yale University School of Medicine. Dr. High was named as a Howard Hughes Medical Institute Investigator in 2003 and is also an elected member of both the Institute of Medicine and the American Academy of Arts & Sciences. She is a member and former Councillor of ASH, a former president of the American Society of Gene and Cell Therapy, and a member of the International Society of Thrombosis and Hemostasis.
"Dr. High is unquestionably a leader in gene therapy and has made remarkable, ground-breaking contributions to the field, demonstrating creativity, determination, and the ability to overcome numerous obstacles," said ASH President Janis L. Abkowitz, MD, of the University of Washington. "Through her countless discoveries, Dr. High has transformed the notion of utilizing genetically engineered mechanisms for treatment of incurable inherited disorders from a distant vision to reality."
The American Society of Hematology (ASH) (http://www.