Long-Term Factor IX Expression and Improved Blood Clotting Time Achieved
SEATTLE, WASHINGTON (May 28, 1998): Scientists at Avigen, Inc. (Nasdaq: AVGN), and The Children's Hospital of Philadelphia have developed a gene therapy approach for the treatment of hemophilia B using a single intramuscular administration of an adeno-associated virus (AAV) vector containing the gene for coagulation factor IX. Katherine A. High, M.D., a hematologist at The Children's Hospital of Philadelphia and Avigen scientific advisor, will present research at the inaugural meeting of the American Society of Gene Therapy that shows the ability of AAV-mediated gene therapy to achieve sustained production of factor IX resulting in improved blood clotting times in a large animal model of hemophilia B.
"These results are very exciting, and can validate that AAV vectors in this model can provide sustained and long-term expression--something that other gene therapy systems have had limited success demonstrating," said John Monahan, Ph.D., president and chief executive officer of Avigen, Inc. "Based on these results and additional R&D advances at Avigen, we are working closely with Dr. High to develop this therapy further with the objective of initiating a human clinical trial as early as the end of calendar 1998 which, if successful, will represent a major advance in the treatment of hemophilia B."
"Our work extends previous results obtained in mice to a well-defined, naturally occurring, large animal model of hemophilia B that is very similar to the disease in humans," commented Dr. High. "This is the first example of the intramuscular administration of a gene therapy vector resulting in sustained improvement of the clotting abnormality in hemophilia and is an important step in justifying this approach for the treatment of patients with hemophilia B."
A single intramuscular administration of an AAV vector containing the gene for
factor IX was given. The animals showe
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