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Contact: Kathryn Ryan, 914-740-2100,
Katherine High Discusses Progress and Challenges in Gene Therapy for Hemophilia and Inherited Retinopathies

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,"

About the Journal

Human Gene Therapy, the Official Journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies, is an authoritative peer-reviewed journal published monthly in print and online. Led by Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, Human Gene Therapy presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to gene transfer in humans. Its companion journals, Human Gene Therapy Methods, published bimonthly, focuses on the application of gene therapy to product testing and development, and Human Gene Therapy Clinical Development, published quarterly, features data relevant to the regulatory review and commercial development of cell and gene therapy products. Tables of contents for all three publications and a free sample issue may be viewed on the Human Gene Therapy website.

About the Publisher

Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Nucleic Acid Therapeutics, Tissue Engineering, Stem Cells and Development, and Cellular Reprogramming. Its biotechnology trade magazine, GEN (Genetic Engineering & Biotechnology News), was the first in its field and is today the industry’s most widely read publication worldwide. A complete list of the firm’s 80 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.