Lentigen Corporation today announced an exclusive licensing agreement with Case Western Reserve University (CWRU) in Cleveland, Ohio, to develop a novel stem-cell therapy for glioblastoma, an aggressive type of brain cancer. Under the agreement, Case Western and its primary affiliate, University Hospitals Case Medical Center, will lead the first in vivo clinical trial, and Lentigen will be responsible for further clinical development and commercialization.

The new therapy will be based on MGMT, a gene that has been shown to repair damaged DNA. A specially designed lentiviral vector will be used to deliver the gene specifically to bone-marrow stem cells that have been damaged by drugs commonly used to treat cancer. A Phase I clinical trial, which will be held at Case Western later this year, will evaluate the potential of this lentiviral vector-MGMT technology (LV-MGMT) to enhance current treatments for glioblastoma by repairing and preventing this damage to bone-marrow stem cells.

"This clinical trial will represent the first time lentiviral vectors will be used with MGMT for stem cell therapy in humans," said Stanton L. Gerson, MD, director of the Ireland Cancer Center of University Hospitals Case Medical Center and the Case Comprehensive Cancer Center, who co-developed the technology with Anthony E. Pegg, Ph.D., the Evan Pugh Professor of Cellular and Molecular Physiology, and Pharmacology at Penn State University. Dr. Gerson commented, "Glioblastoma is a serious diagnosis with limited treatment options, so this new therapeutic approach has the potential to greatly enhance options for patients with this terrible disease."

Boro Dropulic, Ph.D., Lentigen's founder, president and chief scientific officer, commented, "LV-MGMT technology has the potential to greatly enhance the therapeutic efficacy of existing drugs that target glioblastoma by lowering their toxicity to bone marrow stem cells. Beyond glioblastoma, this technology could also be used for therapies for other diseases such as AIDS, leukemia and hemophilia. We are quite excited to be working with Dr. Gerson to move LV-MGMT into the clinic."

Tim Ravenscroft, Lentigen's CEO, commented, "We feel very fortunate to be collaborating with Dr. Gerson to develop this promising technology with broad potential applications. Our exclusive license to develop this technology is a significant step towards our goal of applying our lentiviral vector expertise to the development of cutting-edge biotherapeutics."

About Lentiviral Vectors

Lentiviral vectors (LV) are vehicles that can deliver genes or RNAi into cells with up to 100% efficiency and stability. By comparison, other viral vector systems such as non-viral, adenoviral and adeno-associated viral vectors have not been shown to achieve both high and stable gene delivery in dividing cells.

Gene delivery is accomplished by the binding and fusing of the LV pseudotyped envelope protein to the target cell membrane. The LV RNA containing the gene or gene silencing sequence is then incorporated into the cell via reverse transcription creating a DNA complex. This complex enters the nucleus incorporating into the chromosomal DNA creating a stable molecule. The gene sequence is integrated in the chromosome and is copied along with the DNA during ongoing cell division.

About Lentigen Corporation

Lentigen Corporation is a privately owned biotechnology company focused on the development of lentiviral vector technology for a wide range of therapeutic, vaccine and research applications in biotechnology and medicine. 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. Partnerships include The National Institutes of Health, The University of Pennsylvania, ThermoFisher Scientific and The U.S. Army. For further information, visit . /