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Is the first commercial gene therapy product within sight?

Mary Ann Liebert, Inc./Genetic Engineering News

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IMAGE: 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... view more

Credit: ©Mary Ann Liebert, Inc., publishers

New Rochelle, NY, October 15, 2015--Highly anticipated phase III clinical trial results of Spark Therapeutics's gene therapy to treat visual impairment are due by year-end and could have profound implications for the broader gene therapy field, leading to the first approved product in the U.S. A detailed analysis of the design and use of SPK-RPE65 to treat Leber congenital amaurosis type 2 (LCA2), the possibility of expanding its use to other patient populations, and the anticipated impact of a first commercial gene therapy product is explored in Human Gene Therapy Clinical Development, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Human Gene Therapy Clinical Development website until November 15, 2015.

Joshua Schimmer and Steven Breazzano, Piper Jaffrey & Co., New York, NY, predict the likelihood that the SPK-RPE65 phase III data will meet the primary goal of improving patients' ability to see and function at night or in dimmer light levels. Secondary endpoints include enhanced visual acuity and peripheral field light sensitivity or an effect on the underlying degenerative process.

In the article "Investor Outlook: Focus on Upcoming LCA2 Gene Therapy Phase III Results," the authors examine the science supporting a gene therapy approach to treating LCA2 in which an adeno-associated viral vector delivers the RPE65 gene (which is mutated in LCA2) to the eye via subretinal injection. At least 10 other biotechnology companies have gene therapy products in development targeting retinal diseases.

"Josh and Steve have done a wonderful job summarizing this potential milestone," says Human Gene Therapy Clinical Development Editor James M. Wilson, MD, PhD, Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA.

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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 the 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, Genetic Engineering & Biotechnology News (GEN), 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.

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