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 ALAMEDA, Calif., Sept. 29 /PRNewswire/ -- Avigen, a biopharmaceutical company focusing on gene therapy products utilizing adeno-associated virus (AAV) vectors, announced today that it has received exclusive worldwide rights to a novel multidrug resistance gene from PARTEQ. The gene, which was developed at Queen's University at Kingston, Ontario, Canada, and for which a patent application is pending, expresses a multidrug resistance-associated protein (MRP) that causes cells to become tolerant to otherwise toxic levels of cytotoxic drugs.
 PARTEQ, the technology transfer arm of Queen's University, licensed the gene to Avigen for use with the company's proprietary AAV vectors. In return, PARTEQ will receive from Avigen an issue fee, milestone and maintenance payments and a royalty based on net sales of products developed with the licensed technology.
 The MRP gene was first discovered as a result of a research effort headed by Susan Cole and Roger Deeley of Queen's University. The gene's protein product, MRP, is a member of a superfamily of energy-dependent transport proteins that may function as a pump ridding the cell of toxic substances. Increased levels of MRP have been found in multidrug- resistant human lung cancer cell lines and other types of cancer cells whose resistance cannot be explained by any other drug resistance gene.
 Avigen's AAV vectors will be employed to transfer the gene into normal bone marrow cells of patients undergoing chemotherapy. The genetically modified cells will then be injected back into the patient. The company expects this gene therapy procedure to protect healthy bone marrow cells and their progeny from destruction, allowing for more aggressive and potentially more effective treatment with chemotherapeutic drugs.
 "We are excited about the combined potential of this technology with our AAV vectors and plan to optimize their use in our chemotherapy drug tolerance program," said John Monahan, Ph.D., president and chief executive officer of Avigen. "Because of their inherent non-pathogenic and targeted cell expression characteristics, our vectors may provide for the most efficient transfer of these genes. The expected result will be increased chemotherapy tolerance of healthy cells, allowing for higher levels of toxic drugs to attack and kill cancer cells."
 AAV is nonpathogenic and has never been associated with disease even though greater than 85 percent of the U.S. population is seropositive for the virus. Avigen's AAV vector gene delivery system, unlike the more commonly explored retrovirus vectors, is stable upon storage at room temperature, is not inactivated by components in the blood when directly injected into a patient and exhibits targeted cell expression without the risks associated with other virus vector systems.
 One of the leading weapons in fighting cancer, chemotherapeutic drugs also are highly toxic and produce side effects which limit their therapeutic use. Rapidly dividing cells such as those of the hematopoietic system are particularly sensitive to these drugs and often are destroyed during chemotherapy treatments -- resulting in anemia and infections.
 Avigen, located in Alameda, is a privately held biopharmaceutical company committed to the development of gene therapy products that are practical and easy to administer. Through its proprietary technology which utilizes adeno-associated virus (AAV) vectors for delivering therapeutic genes to patients' cells, the company intends to reduce the complexity of gene therapy while increasing the safety, effectiveness and commercial viability of gene therapy products. Avigen is initially focused on treatments for hemoglobin disorders, certain viral infections and chemotherapy drug tolerance.
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 /CONTACT: Wanda de Vlaminck of Avigen, 510-748-7153; or Sylvia Wheeler of Russell-Welsh Inc., 415-312-0700, for Avigen/

CO: Avigen ST: California IN: MTC SU:

LH-TM -- SF002 -- 6668 09/29/93 09:00 EDT
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Publication:PR Newswire
Date:Sep 29, 1993

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