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Avigen Demonstrates Gene Delivery Success With Erythropoietin

ALAMEDA, Calif., Nov. 26 /PRNewswire/ -- Avigen, Inc. (Nasdaq: AVGN) today announced the successful results of a study to demonstrate the utility of gene therapy for the long term expression of therapeutic proteins. In a report published in the November 26, 1996 issue of Proceedings of the National Academy of Science, Avigen scientists demonstrated the sustained production of the human protein erythropoietin (Epo) in mice for nine months following the single injection of its proprietary adeno-associated virus (AAV) vector containing the gene for erythropoietin.

"This result is an important step in demonstrating the practical utility of gene therapy in treating a variety of medical indications," said John Monahan, Ph.D., President and Chief Executive Officer of Avigen. "While the results with Epo are very exciting, the implications for the use of AAV vectors in achieving the sustained expression of other therapeutic proteins is even more intriguing."

Erythropoietin is a protein produced by the kidney that stimulates the bone marrow to produce red blood cells. The body's production of this protein can be dramatically, even totally, lost through kidney disease, and renal failure.

In the study, performed by Avigen in collaboration with scientists at The Johns Hopkins School of Medicine, AAV-Epo vector produced by Avigen was administered as a single dose into the muscle of mice. Treated animals showed a dose-related increase in human erythropoietin levels in their serum as well as an increase in the number of red blood cells. Following administration of the AAV vector, levels of Epo increased steadily over a six week period and subsequently remained stable for at least an additional eight months.

The study indicated that the use of the AAV-Epo vector resulted in significantly greater levels of Epo following intramuscular administration than those observed following injection of a plasmid DNA vector. "The striking observation in this work is the enhanced efficiency of AAV vectors over plasmid DNA vectors in leading to sustained high level expression of a therapeutic gene," stated Barry J. Byrne, M.D., Ph.D., Assistant Professor of Pediatrics and Pathology at The Johns Hopkins School of Medicine and co-author of the report.

"The observation that the single administration of the Epo gene delivered in an AAV vector results in a sustained biologic effect highlights the potential of the AAV vector system and of gene therapy," said Gary J. Kurtzman, M.D., Vice President of Research and Development at Avigen and co-author of the report. "We are currently extending these efforts to other clinically important therapeutic proteins."

Avigen scientists will report on progress in the Company's research and development efforts on December 8, 1996 at the American Society of Hematology Meeting in Orlando, Florida.

Avigen, Inc. is a biotechnology company involved in the development of gene therapy products derived from adeno-associated virus for the treatment of inherited and acquired diseases. The Company's proposed gene therapy products are designed for in vivo administration to achieve the production of therapeutic proteins within the body. Avigen is focusing its efforts on developing products to deliver genes for the treatment of brain, liver and prostate cancer, anemia, hemophilia, hyperlipidemia, metabolic storage diseases and blood cell-related diseases, including sickle cell anemia, beta-thalassemia and HIV.

Except for the historical information contained herein, this news release contains forward-looking statements that involve risks and uncertainties. The Company's actual results may differ materially from those discussed herein. Factors that could cause or contribute to such differences include, but are not limited to, those discussed in this press release, in the Company's Annual Report on Form 10-K for the year ended June 30, 1996 and in the Company's Registration Statement No. 333-16175.

SOURCE Avigen Inc.
 -0- 11/26/96

/CONTACT: Thomas J. Paulson, Chief Financial Officer of Avigen Inc., 510-748-7150/


CO: Avigen Inc. ST: California IN: MTC SU:

RN-TC -- SFTU028 -- 4170 11/26/96 08:02 EST
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Publication:PR Newswire
Date:Nov 26, 1996
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