Sangamo Biosciences Inc (SGMO)

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4:34PM Sangamo BioSci announces presentation of data demonstrating a disruptive and broadly leverageable platform for protein replacement therapies at ASH meeting (SGMO) 6.06 +0.13 : Co announced the presentation of new pre-clinical data demonstrating the successful application of its In Vivo Protein Replacement Platform. Based on Sangamo's zinc finger DNA-binding protein (ZFP) genome-editing technology, the platform 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 diseases (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. The data were presented at the 54thAnnual Meeting of the American Society of Hematology (ASH) which is being held in Atlanta.