Positive clinical trial results on gene transfer to treat macular degeneration

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, 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,… view more

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New Rochelle, NY, Oct.20, 2016 –The long-term safety of gene transfer to treat neovascular age-related macular degeneration (NVAMD), and the production of two therapeutic proteins encoded by those genes for at least 2.5 years in the eyes of patients with advanced NVAMD are demonstrated in the results of a clinical trial published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Human Gene Therapy website.

In the article entitled "Lentiviral Vector Gene Transfer of Endostatin/Angiostatin for Macu-lar Degeneration (GEM) Study," Peter Campochiaro, MD and coauthors from The Wilmer Eye Institute, Johns Hopkins University School of Medi-cine (Baltimore, MD), Casey Eye Institute, Oregon Health & Science University (Portland, OR), University of Iowa Hospitals and Clinics (Iowa City), and Oxford BioMedica (Oxford, U.K.) present the design and results of the Phase 1 clinical trial. Each study participant re-ceived treatment in one eye consisting of a subretinal injection of a viral vector engineered to deliver two therapeutic genes, endostatin and angiostatin, each of which blocks the for-mation of blood vessels to combat the progression of NVAMD.

"It has been the hope that the spectacular success of gene therapy for genetic retinal disor-ders would translate into a platform for treating more common diseases of the eye," says Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Edu-cation and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, Worcester, MA. "Dr. Campochiaro's pioneering work on age-related macu-lar degeneration (AMD) demonstrates the feasibility of such an approach for one of the most common vision-threatening disorders, and one whose incidence is rising dramatically as our population ages."

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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, 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.

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