GAITHERSBURG, Md., Feb.13, 2013 /PRNewswire/ --Lentigen Corporation, a biotechnology company specializing in the development and manufacture of lentiviral gene delivery products, announced today that that the U.S. Food and Drug Administration (FDA) has granted orphan drug status to P140K methylguanine methyltransferase (MGMT) transduced human CD34 cells (product name: LG631-CD34) for bone marrow protection in the treatment of glioblastoma multiforme. Orphan drug designation qualifies Lentigen for seven years of market exclusivity following marketing approval by the FDA and provides other development-related incentives.

"We are very pleased that the FDA has granted our brain cancer product orphan drug status," said Tim Ravenscroft CEO of Lentigen Corporation. "The market exclusivity which this designation provides, together with our intellectual property rights, gives us increased impetus to advance the development of this product, enabling us to play our part in improving the lives of patients with glioblastoma."

"LG631-CD34 consists of the patients' adult hematopoietic stem cells genetically modified with a Lentiviral vector expressing a human MGMT gene variant, which is designed to protect the cells from the toxic side-effects of Temodar*, a standard of care treatment for glioblastoma multiforme" said Dr. Boro Dropulic, the Chief Scientific Officer of Lentigen Corporation. "Protection of blood-forming hematopoietic stem cells from the side-effects effects of Temodar would provide immediate benefits to patients. It potentially enables higher doses and more intensive drug treatment with reduced toxicity, resulting in improved clinical outcomes.

LG631-CD34 is currently being evaluated in a NIH grant-funded Phase I clinical trial (http://www.clinicaltrials.gov/ct2/show/NCT01269424) at University Hospitals (UH) Case Medical Center under the leadership of Stanton Gerson, MD, Director of the Seidman Cancer Center at UH Case Medical Center, Director of the National Center for Regenerative Medicine and Case Comprehensive Cancer Center and Asa, Patricia Shiverick - Jane Shiverick (Tripp) Professor of Hematological Oncology at Case Western Reserve University School of Medicine and Andrew Sloan MD, Associate Professor & Vice-Chairman, Department of Neurological Surgery and the Peter D. Cristal Chair of Neurosurgical Oncology at Case Western Reserve University School of Medicine, Director, Brain Tumor and Neuro-Oncology Center, UH Case Medical Center and a member of the Case Comprehensive Cancer Center.

"Glioma is such an aggressive and challenging cancer," said Dr. Gerson. "When patients are diagnosed with this life threatening disease, they have an average life expectancy of less than 12 months. The medical community needs to find new treatment strategies that can improve clinical outcomes in this devastating disease. I am hopeful that LG631-CD34 can make an important contribution and help us improve the lives of our patients."

Dr. Stanton Gerson is an inventor on this mutant alkyltransferase technology. He, as well as Case Western Reserve University and University Hospitals, could gain from commercialization of the invention.

The clinical trial is supported by Grant Number R42CA128269 from the National Cancer Institute and a grant to the Center for Stem Cell & Regenerative Medicine (CSCRM) from Ohio's Third Frontier Commission under its Research Commercialization Program. The content of this news release is solely the responsibility of the authors and does not necessarily represent the official views of the National Cancer Institute or the National Institutes of Health.

The U.S. Orphan Drugs Act aims to encourage the development of drugs involved in the diagnosis, prevention or treatment of a medical condition affecting fewer than 200,000 people in the United States. Orphan drug designation grants U.S. market exclusivity to a drug for a particular indication for a seven-year period if the sponsor complies with certain FDA specifications. Additional incentives for the sponsor include tax credits related to clinical trial expenses and a possible exemption from the FDA user fee, and the orphan status also allows the sponsor to apply for grants to support clinical trials.

About Lentigen CorporationLentigen Corporation is a privately owned biotechnology company focused on the development of lentiviral vector technology for a wide range of therapeutic, vaccine, and bioproduction applications. Lentiviral vectors are the most efficient vehicles for the delivery of genes or gene silencing sequences stably into cells. Lentigen is a highly collaborative company, co-developing Lentiviral vector-based products across a broad spectrum of bench to clinical applications. Collaborations include The National Institutes of Health, IAVI, Harvard Medical School, Expression Therapeutics, Scripps Institute, Case Western Reserve University, Loyola University Chicago, The University of Pittsburgh, and the University of Minnesota. Lentigen is primarily funded by Burrill Capital Fund IV, L.P. (San Francisco, CA). For further information, visit http://www.lentigen.com.

About Case Comprehensive Cancer Center Case Comprehensive Cancer Center is an NCI-designated Comprehensive Cancer Center located at Case Western Reserve University. The center, now in its 25th year of funding, integrates the cancer research activities of the largest biomedical research and health care institutions in Ohio Case Western Reserve, University Hospitals (UH) Case Medical Center and the Cleveland Clinic. NCI-designated cancer centers are characterized by scientific excellence and the capability to integrate a diversity of research approaches to focus on the problem of cancer. It is led by Stanton Gerson, MD, Asa and Patricia Shiverick- Jane Shiverick (Tripp) Professor of Hematological Oncology, director of the National Center for Regenerative Medicine, Case Western Reserve, and director of the Seidman Cancer Center at UH Case Medical Center.

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Lentigen Receives U.S. Orphan Drug Designation for Novel Gene Therapy for glioblastoma multiforme

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