The technology was developed through the collaborative effort of scientists from Avigen and Dr. Katherine High at The Children s Hospital of Philadelphia. We believe that the successes of this past year will provide the foundation for Avigen s future growth and development. The Company continues to maintain its sharp product focus as it builds on the scientific and managerial foundation in order to develop gene therapy solutions for patients whose conditions have not been successfully treated by conventional therapeutic approaches. These disorders represent attractive commercial opportunities for the application of Avigen s technology. It affects 10,000 to 20,000 patients, who suffer from anemia, bone damage, and enlarged livers and spleens.
Current treatment aimed at replacing the missing protein is inadequate and impractical.
There is no effective therapy. Avigen has succeeded in producing high levels of circulating EPO for more than one year following injection into mouse muscle of the AAV vector with the human EPO gene and has shown therapeutic effects in an animal model of beta-thalassemia. This research will also serve as a precursor for a factor VIII trial.