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Katherine High talks gene therapy progress for hemophilia & inherited retinopathies

Mary Ann Liebert, Inc./Genetic Engineering News

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IMAGE: Human Gene Therapy, Human Gene Therapy Methods, and Human Gene Therapy Clinical Development provide complete, multidisciplinary coverage on all aspects of gene therapy. view more

Credit: ©2017 Mary Ann Liebert, Inc., publishers

New Rochelle, NY, January 4, 2017--Gene therapy has shown some of its most promising early results in treating patients with hemophilia and inherited retinal disorders that cause vision loss and blindness, both important research and drug development targets during the career of Katherine High, MD, President and Chief Scientific Officer of Spark Therapeutics (Philadelphia, PA). James M. Wilson, MD, PhD, Editor of Human Gene Therapy Clinical Development, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers recently spoke with Dr. High and the absorbing interview is published in the Journal. The article is available free on the Human Gene Therapy Clinical Development website until February 4, 2017.

The article "Interview with Katherine A. High, MD" explores a range of topics, including Dr. High's long career in gene therapy and her decision to study hemophilia as a model for in vivo and clinical gene therapy. Dr. Wilson expands the conversation to focus on the work underway at Spark Therapeutics, which is focusing on not only the clinical development of gene therapy for hematologic disorders, but also for inherited retinal diseases and neurodegenerative diseases. Dr. High discusses the progress in each of these programs and comments on key scientific, regulatory, and commercial challenges.

"In this interview, Kathy provided fascinating insight into her career and the key decisions she made in advancing in vivo gene therapy across multiple fronts," says Human Gene Therapy Clinical Development Editor James M. Wilson, MD, PhD, Director of the Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA. "Her leadership in both the academic and business domains has been critical to the current success that the field is realizing."

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