Gene therapy is a 'disruptive science' ready for commercial development

New Rochelle, NY, January 24, 2012—The time for commercial development of gene therapy has come. Patients with diseases treatable and curable with gene therapy deserve access to the technology, which has demonstrated both its effectiveness and feasibility, says James Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy in a provocative commentary and accompanying videocast. Human Gene Therapy and Human Gene Therapy (HGT) Methods are peer-reviewed journals published by Mary Ann Liebert, Inc..

Until recently, gene therapy has been reserved for severe diseases with few treatment options. But the recent report of its successful use to treat hemophilia B, which would offer patients a therapeutic alternative that could replace the need for regular, lifelong protein replacement infusions, has brought gene therapy to the forefront as a technology capable of competing with and disrupting traditional forms of treatment. Although gene therapy for hemophilia B is still in early-stage clinical testing, a similar approach is in development to treat hemophilia A, and together these life-threatening diseases represent a $6.5 billion market for current protein replacement therapies.

The technical feasibility of gene therapy "has been established in multiple diseases and with different technology platforms," says Dr. Wilson, in the Commentary "It's Time for Gene Therapy to Get Disruptive!" He predicts that "2012 will usher in an era of commercial development of gene therapy that, although likely to begin slowly, will quickly gather momentum."

"The scientific community has been promising for years that disruptive change would follow from investments in biomedical research, such as the doubling of NIH and the sequencing of the human genome," says Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education Dean, Provost, and Executive Deputy Chancellor, Gene Therapy Center, and Departments of Pediatrics and Microbiology & Physiologic Systems, University of Massachusetts Medical School. "Dr. Wilson's comments strike at the heart of fulfilling that promise. Now that the technology is working, the next phase is to develop commercially viable models for gene therapy in the health care marketplace, so that these therapies can be delivered to the patients who need them."

Human Gene Therapy, the Official Journal of the European Society of Gene and Cell Therapy, British Society for Gene 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 that 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. Tables of content and a sample issue may be viewed online at www.liebertpub.com/hum.

Mary Ann Liebert, Inc. 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, 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 70 journals, books, and newsmagazines is available at our website www.liebertpub.com.

Mary Ann Liebert, Inc. 140 Huguenot St., New Rochelle, NY 10801-5215 www.liebertpub.com
Phone: (914) 740-2100 (800) M-LIEBERT Fax: (914) 740-2101

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