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Contact: Jennifer Gatti, 914-740-2100,
Pancreatitis Reduced by Nearly 50% After Gene Therapy to Treat Lipoprotein Lipase Deficiency
New Rochelle, NY, November 8, 2016—Over a 6-year period, patients with the genetic disease lipoprotein lipase deficiency (LPLD) who received a single gene therapy treatment of alipogene tiparvovec had a marked reduction in the severity and frequency of pancreatitis. No cases of severe pancreatitis and only one admission to the intensive care unit for an LPLD-related abdominal event were reported in the study 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 until December 8, 2016.

Daniel Gaudet and coauthors from Université de Montréal, Canada, Academic Medical Center and uniQure BV (Amsterdam, the Netherlands), Chiesi Farmaceutici (Parma, Italy), Beaujon Hospital, Denis Diderot University (Paris, France), University Hospital of North Midlands (U.K.), and Erasmus Medical Centre (Rotterdam, the Netherlands) present their findings in the article entitled "Long-Term Retrospective Analysis of Gene Therapy with Alipogene Tiparvovec and Its Effect on Lipoprotein Lipase Deficiency-Induced Pancreati-tis."
The researchers assessed LPLD-related acute abdominal events that required hospital care in a small group of patients treated with a single dose of the gene therapy product Glybera®. The results support an association between gene therapy for this rare genetic disease and overall reductions in healthcare costs and resource utilization.
“As gene therapy moves forward into the mainstream of medicine, it will be critical to de-fine the evidence of its benefit to patients with specific diseases,” says Editor-in-Chief Ter-ence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, Worcester, MA. “This work with alipogene tiparvovec provides an evidence-based guide-line for its use in patients with LPLD going forward.”

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.