Sangamo BioSciences Reveals New Gene Therapy Clinical Development Program For Hemophilia A Treatment

Sangamo BioSciences, Inc. SGMO revealed the presentation of preclinical data supporting the clinical development of its new proprietary gene therapy for hemophilia-A treatment. The company stated that this fresh therapeutic consisted of an adeno-associated virus (AAV) cDNA human Factor 8 (hF8) construct fueled by its proprietary synthetic liver specific promoter.

According to the company, in preclinical trials, it demonstrated a minimum of three times more potent than the current AAV-based cDNA constructs that is currently under evaluation for the treatment of hemophilia A.

Sangamo BioSciences said the data would be presented at the World Federation of Hemophilia (WFH) 2016 World Congress that is being held in Orlando from July 24 – 28. The company said that the data also demonstrated production of supraphysiologic levels of human Factor VIII (hFVIII) in a mouse model of the disease, as well as, in non-human primates (NHPs).

The company's EVP of R&D, Geoff Nichol, commented, "We have compelling preclinical data from this AAV cDNA therapeutic which suggest its potential to be 'best in class' in this highly competitive field. As a leading science and data-driven company, Sangamo has the expertise to efficiently move new technological advances into our therapeutic portfolio."

He added, "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."

On Tuesday, the stock advanced 2.34 percent.

Posted In: BiotechNewsFDAGeneral
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