Avigen And Johns Hopkins Awarded AAV Vectors Patent
The U.S. Patent and Trademark Office has issued Patent No. 5,858,351 to Avigen Inc. and Johns Hopkins University covering the in vivo administration of any gene into skeletal, smooth or cardiac muscle tissue using adeno-associated virus (AAV) vectors. The patent encompasses all methods of making the vector, how the gene is regulated or even what disease is being treated.
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In the case of hemophilia, patients are missing the protein factor IX that causes blood to clot. Avigen scientists have reported long-term expression of clotting factor IX in an animal model of hemophilia B following a single intramuscular (IM) administration of the gene for factor IX utilizing AAV gene therapy, showing therapeutic levels of factor IX more than a year after the initial treatment.
Although the clotting factor is normally produced in the liver, this research indicates that muscle tissue is capable of producing the protein following IM administration of AAV-factor IX gene therapy. "IM administration is a simpler treatment than infusion into the liver,'' says Dr. Mark Kay, director of the Program in Human Gene Therapy and Associate Professor in the Departments of Pediatrics and Genetics at Stanford University School of Medicine and a member of Avigen's Scientific Advisory Board.
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The research underlying the patent was developed through the collaborative effort of scientists from Avigen and Johns Hopkins University. Based in the San Francisco Bay area, Avigen Inc. is a biotechnology company that develops gene therapy products derived from adeno-associated virus for the treatment of inherited and acquired diseases.
"We believe that this is a very significant patent because it encompasses so many disease indications where muscle could serve as a primary or preferred site of administration," said Dr. John Monahan, Avigen's president and CEO. These disease indications include blood diseases such as hemophilia and anemia, cardiovascular diseases such as restenosis, and metabolic storage diseases.
"Muscle administration of the AAV vector is a simple, yet elegant and straightforward approach to gene therapy," according to Dr. Monahan, "as opposed to the more invasive method of injecting the vector into an organ, such as the liver."
This is the second patent relating to adeno-associated virus (AAV) Avigen has been issued in the past six weeks and complements its recently issued Patent No. 5,846,528, broadly covering erythropoietin gene therapy
Avigen scientists have observed long-term expression of erythropoietin following a single administration of AAV erythropoietin into skeletal muscle of normal and anemic mice. Nearly two years after administration, the mice are exhibiting therapeutic levels of EPO, a protein produced by the kidney that stimulates cells in the bone marrow to produce red blood cells; it is also involved in the production of hemoglobin.
In inherited disorders such as sickle cell anemia and beta-thalassemia, the body produces inadequate levels of, or abnormal, hemoglobin molecules. Acquired anemias occur in patients with renal failure, AIDS, and in those receiving chemotherapy for cancer, when the body's production of red blood cells is inadequate.
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