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

Scientists Present Preclinical Data Demonstrating High Levels of Factor VIII Protein Expression from Proprietary Therapeutic AAV cDNA Construct at World Federation of Hemophilia 2016 World Congress

Company's Goal is to File Investigational New Drug Application for New Factor VIII Gene Therapy Approach in 2016

RICHMOND, Calif., July 26, 2016 /PRNewswire/ -- Sangamo BioSciences, Inc. (NASDAQ: SGMO), the leader in therapeutic genome editing, today announced the presentation of preclinical data that supports the clinical development of its new proprietary gene therapy for the treatment of hemophilia A. This new therapeutic comprises an adeno-associated virus (AAV) cDNA human Factor 8 (hF8) construct driven by Sangamo's proprietary synthetic liver specific promoter, which in preclinical studies is at least three times more potent than existing AAV-based cDNA constructs currently under evaluation for the treatment of hemophilia A.

"We have compelling preclinical data from this AAV cDNA therapeutic which suggest its potential to be 'best in class' in this highly competitive field," said Geoff Nichol, M.B., Ch.B., Sangamo's executive vice president of research and development. "As a leading science and data-driven company, Sangamo has the expertise to efficiently move new technological advances into our therapeutic portfolio. We intend to move this cDNA gene therapy approach for hemophilia A into the clinic as soon as possible while continuing to optimize our in vivo zinc finger nuclease (ZFN) genome editing approach, which may be more suitable for certain groups of hemophilia A patients."

The data, presented by Sangamo scientists at the World Federation of Hemophilia (WFH) 2016 World Congress, being held in Orlando, 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 1012 vgs/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.

"As we continue our evolution from a platform company to a clinical-stage therapeutic...


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