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Sangamo BioSciences Presents New Data From In Vivo Protein Replacement Platform For Development Of ZFP Therapeutics® For Monogenic Diseases

Stocks in this article: SGMO SHP SHPG

RICHMOND, Calif., May 20, 2013 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced the presentation of new data demonstrating the successful application of Sangamo's proprietary In Vivo Protein Replacement Platform (IVPRP) to produce therapeutically relevant levels of Factor VIII in a mouse model.  Sangamo has partnered with Shire AG (LSE: SHP, NASDAQ: SHPG) to develop ZFP Therapeutics for both hemophilia A and B using this approach. The data were presented at the 16 th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) which was held in Salt Lake City from May 15-18, 2013.


"Our IVPRP provides a potentially curative therapeutic solution for hemophilia A – a monogenic disease caused by the absence of the coagulation protein, Factor VIII," said Philip Gregory. D. Phil., Sangamo's vice president of research and CSO. "These data provide further proof-of-concept for the broad application of our IVPRP strategy for monogenic diseases currently treated using protein replacement.  Our data demonstrate that a single systemic treatment results in the stable production of therapeutically relevant levels of functional Factor VIII."

Hemophilia A, the most common form of the bleeding disorder, is caused by a genetic defect in the gene encoding Factor VIII, a blood clotting factor.  Sangamo's IVPRP approach enables the precise insertion of a replacement Factor VIII gene into the genome at what is known as a "safe harbor site." Specifically, Sangamo's IVPRP uses the albumin gene as the site of insertion ensuring that Factor VIII will be highly expressed at stable levels exclusively in the liver.

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. 

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