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Sangamo BioSciences (SGMO) Data Presented at ASH Bolsters ZFN Platform - Wedbush

December 7, 2015 2:58 PM EST
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Wedbush analyst Liana Moussatos reiterating an Outperform rating and $30 price target on Sangamo BioSciences (NASDAQ: SGMO) saying preclinical data on its Hemophilia B program at ASH bolsters its ZFN platform.

Moussatocommented, "With the recent acceptance of its IND application for its SB-FIX/Hemophilia B program, Sangamo presented non-human primate (NHP) data showing that ZFNs can be used to insert a corrective human factor IX gene into the albumin locus in liver cells. A single intravenous administration of SB-FIX, resulted in > 3% of normal factor IX production in NHP, an amount that may be able to ameliorate symptoms of the disease. Moreover, because of the relatively high expression from the albumin promoter, it may be possible to provide therapeutic levels of factor IX protein via expression from a relatively small percentage of cells. Importantly, it was shown that ZFN targeting was specific, with only a single off-target site (SMCHD1) observed when SB-FIX was given at levels much higher than potential clinical doses. In both NHP and mice, genome editing was well tolerated with no noticeable impact on circulating albumin levels. We anticipate Sangamo will begin clinical testing of SB-FIX for Hemophilia B in H1:16."

For an analyst ratings summary and ratings history on Sangamo BioSciences click here. For more ratings news on Sangamo BioSciences click here.

Shares of Sangamo BioSciences closed at $9.14 yesterday.



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