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Sangamo Biosciences Inc Announces New Data From In Vivo Protein Replacement Platform For Development Of ZFP Therapeutics For Monogenic Diseases


Monday, 20 May 2013 07:00am EDT 

Sangamo Biosciences Inc announced the presentation of new data demonstrating the successful application of Sangamo's In Vivo Protein Replacement Platform (IVPRP) to produce therapeutically relevant levels of Factor VIII in a mouse model. Sangamo has partnered with Shire AG to develop ZFP Therapeutics for both hemophilia A and B using this approach. Based on Sangamo's zinc finger DNA-binding protein (ZFP) genome-editing technology, the IVPRP enables the permanent production of therapeutic proteins from the liver with a single systemic treatment, potentially providing curative treatments for a range of monogenic diseases including hemophilia and lysosomal storage disorders (LSD) such as Gaucher and Fabry disease. Such diseases are currently treated by regular infusions of protein or enzyme replacement therapy (ERT) throughout the patient's life. Data were presented on the production of Factors VIII and IX for the correction of hemophilia A and B, as well as four enzymes absent in a panel of LSDs by Sangamo's collaborators from the laboratory of Katherine A. High, M.D., director of the Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia, and Investigator, Howard Hughes Medical Institute. 

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30 Jul 2014