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Sangamo BioSciences Announces New Gene Therapy Clinical Development Program for Treatment Of Hemophilia A

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Additional Relevant Details The data, presented by Sangamo scientists at the World Federation of Hemophilia (WFH) 2016 World Congress, being held inOrlando, Florida, from July 24 - 28, 2016, demonstrated production of supraphysiologic levels of human Factor VIII (hFVIII) in a mouse model of the disease and in non-human primates (NHPs). Mean levels of hFVIII several fold in excess of normal, obtained using research grade AAV in NHPs, were confirmed by dosing with AAV manufactured using Sangamo's clinical process. In these animals, mean hFVIII levels ranged from 5 - 230% of normal and were obtained using AAV doses in the 6 x 1011 - 6 x 1012vgs/kg range - the most potent dose response in NHPs thus far disclosed for an hF8 cDNA. Based on other studies in the field, vector dose and corresponding hFVIII expression levels observed in NHPs are highly predictive of those observed in patients. The high potency of this novel therapeutic may enable clinically relevant levels of hFVIII to be obtained using lower vector doses, which potentially provides a better therapeutic risk/benefit profile for patients. Sangamo is conducting additional studies to determine the minimal effective dose necessary to provide therapeutic benefit.
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Jul 26, 2016
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Related Keywords New Gene Therapy, Hemophilia A, Cdna, Zinc Finger Nuclease Genome