AAV vectors are capable of efficient in vivo gene transfer to a number of tissues including muscle, liver, and the nervous system. AAV-mediated expression of secreted proteins in the muscle and liver can result in significant levels of protein in the bloodstream. The duration of gene expression in these tissues following a single administration of a vector can be maintained for periods of months or even years. These properties, together with a favorable toxicity profile and minimal immunogenicity, indicate that AAV is likely to be a suitable vector for gene therapy applications which require sustained delivery of a therapeutic protein over long periods of time. Avigen is investigating the use of AAV vectors to deliver the factor IX gene for the treatment of hemophilia B and the factor VIII gene for hemophilia A. Preclinical efficacy and safety studies in several animal species including the canine hemophilia B model have provided the basis for a phase I clinical trial of Coagulin B, an AAV vector containing the factor IX gene administered intramuscularly. This trial is currently underway at Children's Hospital of Philadelphia and at Stanford University. The presentation will provide an update on Avigen's programs for both hemophilia A and B.
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