Date: Thu, 18 Dec 1997 17:52:19 GMT Server: Apache/1.2.4 Last-Modified: Thu, 30 Oct 1997 19:28:55 GMT ETag: "9762-1158-3458dff7" Content-Length: 4440 Accept-Ranges: bytes Connection: close Content-Type: text/html Anemia

Anemia

Anemia is a blood disorder that results in a deficiency of red blood cells and hemoglobin. This therapy is in preclinical development. There are two broad categories of anemic diseases: inherited and acquired. In inherited diseases, such as sickle cell anemia and beta-thalassemia, the anemia is caused by abnormal or inadequate hemoglobin molecules. Acquired anemia can be caused by renal failure, AIDS or chemotherapy for cancer. In these cases, the body does not produce an adequate amount of hemoglobin.

The current standard of care treatment for patients with acquired anemia is erythropoietin (EPO) injections. EPO is a glycoprotein hormone that stimulates the production of red blood cells in the bone marrow. Currently, Amgen manufactures a recombinant form of erythropoeitin, for which it has the rights to market in the United States for renal dialysis patients, while Johnson & Johnson has the rights to market EPO for all other indications. There are an estimated 215,000 renal failure patients in the United States that receive dialysis, with approximately 88% of them using Epogen. About 50,000 AIDS patients also use EPO. Total sales of erythropoietin (through both Amgen and Johnson & Johnson) were over $2 billion dollars in 1995. Johnson & Johnson is also in early development stage for small peptides that mimic EPO's receptor binding and may eventually lead to an orally active small molecule EPO substitute drug.

AAV-EPO - Anemia

Avigen has a preclinical program for the treatment of anemia using the AAV vector containing the erythropoietin (EPO) gene. Avigen has shown in animal studies that a single injection of its AAV-EPO vector can provide high levels of EPO in the blood for at least eight months. Moreover, the produces EPO is biologically active, resulting in a continuously elevated hematocrit level in a dose-dependent manner. The company is conducting further efficacy studies in animal models for renal failure and beta thalassemia.

We believe that Avigen's method of increasing EPO levels and red blood cell production has a potential advantage over current treatment. AAV gene therapy offers the possibility of increasing hematocrit to a normal level for an extended time period from a single injection, as compared with the current EPO therapy, which requires two or three injections per week. This may be particularly significant for diseases such as sickle cell anemia and beta thalassemia, where a high concentration of EPO is believed to be required in order to have a therapeutic impact.


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