Sangamo Biosciences, Inc. (NASDAQ:SGMO), a leader in therapeutic genome editing, announced unanimous approval by the National Institutes of Health’s Recombinant DNA Advisory Committee (RAC) of a Phase 1 study protocol for a ZFP Therapeutic as a potential curative therapy for hemophilia B. The Factor IX program will be the first clinical study of in vivo genome editing and the first therapeutic application of Sangamo’s In Vivo Protein Replacement Platform (IVPRP).

Sangamo expects to file an Investigational New Drug (IND) Application with the U.S. Food and Drug Administration (FDA) by the end of 2015 and, pending FDA clearance, plans to initiate a Phase 1 clinical trial in 2016. The Company recently reacquired all of its hemophilia assets from Shire plc as a result of the amendment to the companies’ 2012 collaboration and license agreement.

“Hemophilia B is the first clinical application of our IVPRP which can be applied to many other diseases that are currently treated by protein replacement including hemophilia A and lysosomal storage disorders,” said Geoff Nichol, M.B., Ch.B., Sangamo’s executive vice president of research and development. “Successful review, by the NIH RAC, of the first human in vivo genome editing clinical protocol is a major milestone for Sangamo. We appreciate this careful consideration and unanimous approval of our proposed clinical trial and look forward to the commencement of our Phase 1 study in the near future.”

“We are pleased with the outcome from this thorough review, by the NIH RAC, of our first IVPRP clinical protocol,” said Edward Lanphier, Sangamo’s president and chief executive officer. “We will continue to leverage the potential of this strategy for development of ZFP Therapeutics for other monogenic diseases, and remain on track to file IND applications for the hemophilia B program and the first of our lysosomal storage disorder programs by the end of 2015.” (Original Source)

Shares of Sangamo Biosciences closed yesterday at $6.54. SGMO has a 1-year high of $19.25 and a 1-year low of $6.22. The stock’s 50-day moving average is $8.04 and its 200-day moving average is $12.16.

On the ratings front, Sangamo has been the subject of a number of recent research reports. In a report issued on September 4, Cowen analyst Ritu Baral reiterated a Buy rating on SGMO. Separately, on September 2, Wedbush’s Liana Moussatos reiterated a Buy rating on the stock and has a price target of $30.

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Ritu Baral and Liana Moussatos have a total average return of 19.8% and 20.6% respectively. Baral has a success rate of 49.4% and is ranked #74 out of 3752 analysts, while Moussatos has a success rate of 40% and is ranked #84.

Sangamo BioSciences Inc is a clinical stage biopharmaceutical company. The Company is engaged in the research, development & commercialization of engineered DNA-binding proteins for the development of novel therapeutic strategies for unmet medical needs.