Does the body’s immune response to viral vector delivery systems affect the safety or efficacy of gene therapy?

PUBLIC RELEASE DATE:

8-Jan-2014

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, January 7, 2014Packaging replacement genes in viruses is an effective method to deliver them to target tissues, but the human body mounts an immune response against the virus. The systemic and local immune reactions induced by an adeno-associated virus (AAV)-based gene therapy to treat lipoprotein lipase deficiency, approved for use in Europe, does not affect the safety of gene therapy or expression of the replacement gene for at least one year after delivery, according to a study published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available on the Human Gene Therapy website.

Valeria Ferreira and coauthors, uniQure BV and Academic Medical Center, Amsterdam, the Netherlands, and University of Montreal and Chicoutimi Hospital, Quebec, Canada, evaluated measures of inflammation and adverse clinical events and the expression of a replacement lipoprotein lipase (LPL) gene that was injected intramuscularly into patients with LPL deficiency. The gene was packaged in an AAV vector, as described in the article "Immune responses to intramuscular administration of alipogene tiparvovec (AAV1-LPLS447X) in a phase II clinical trial of Lipoprotein Lipase deficiency (LPLD) gene therapy."

"The clinical data published in this paper were critical to the approval of Glybera," says James Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia. "Furthermore, they provide context for laboratory measurements of immune responses which apparently did not impact product performance."

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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. 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 sister journals are Human Gene Therapy Methods, published bimonthly and focused on the application of gene therapy to product testing and development, and Human Gene Therapy Clinical Development, published quarterly and featuring data relevant to the regulatory review and commercial development of cell and gene therapy products. Tables of content for all three publications and a sample issue may be viewed on the Human Gene Therapy website.