Avigen's Gene Therapy Approach Shows Promise In Treating Hemophilia B With,,Single Intramuscular Admi...( d long-term production of factor IX in ...)

d long-term production of factor IX in a dose-related manner with plasma levels of factor IX being increased for approximately nine months with observations continuing. Researchers observed a partial correction of the whole blood clotting time and partial thromboplastin time, another laboratory test to measure clotting time. Also, there was no toxicity noted in treated animals.

Hemophilia B

Hemophilia B, the second most common severe genetic bleeding disorder worldwide, is caused by an abnormality or deficiency in the blood clotting protein factor IX. To treat or prevent serious bleeding episodes, patients currently receive intermittent or regularly scheduled infusions of factor IX obtained from pooled human plasma or produced by recombinant DNA technology. Such treatments can cost up to $100,000 per year, are inconvenient, and for many individuals do not provide adequate control over their bleeding. Individuals with hemophilia B over time may suffer from spontaneous bleeding episodes which can lead to crippling joint deformities and intracerebral bleeding which can be fatal. Using gene therapy to enable individuals with hemophilia B to manufacture constant levels of factor IX could potentially protect patients from the long-term debilitating effects of their disease, as well as greatly lower the cost of current treatments.

Background on AAV Vectors

Avigen's AAV vectors are derived from adeno-associated virus, a non-pathogenic human virus. The company produces AAV vectors by removing the virus's own genes and replacing them with genes for therapeutic proteins. AAV vectors combine desirable properties of viral and non-viral vectors and may offer potential advantages over other gene therapy vectors. These advantages include efficient delivery of genes to both dividing and non-dividing target cells, absence of viral genes that may be responsible for causing an undesirable immune response, high levels of gene expression, and stability whic
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Contact: Joan Kureczka
JKureczka@aol.com
415-821-2413
Kureczka/Martin Associates
28-May-1998

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