Note: The below is an edited version of an article originally published on PR Newswire. The original release can be read in its entirety here.
Sangamo BioSciences, Inc. 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. The data demonstrated production of supraphysiologic levels of human Factor VIII in a mouse model of the disease and in non-human primates. Sangamo hopes to file an investigational new drug application with the U.S. Food and Drug Administration in 2016.
