Sangamo Biosciences, Inc. (NASDAQ:SGMO), the leader in therapeutic genome editing, announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug application (IND) for SB-913, a zinc finger nuclease (ZFN)-mediated approach designed as a single treatment with the potential to provide a long-lasting therapy for Mucopolysaccharidosis Type II (MPS II, Hunter syndrome). The IND is now active and enables Sangamo to initiate a Phase 1/2 clinical trial (SB-913-1602) to assess the safety, tolerability and potential efficacy of SB-913 in adults with MPS II.

“Our team has successfully prepared and filed three IND applications in short succession, for hemophilia B, MPS I and now MPS II, meeting our stated goal for filing the third IND application for our IVPRP genome editing approach in the first half of 2016,” saidGeoff Nichol, M.B., Ch.B., Sangamo’s executive vice president of research and development. “Our next goal is to continue this momentum and initiate the SB-913-1602 clinical study for MPS II in the second half of 2016. With positive clinical and safety data in adults, our intention with this, and all of our IVPRP programs, is to extend trials into pediatric populations who could benefit most from early intervention with a genome editing approach designed to provide stable expression of therapeutic enzyme throughout the patient’s lifetime.”

“Our gene-based approach is designed to give the patient the ability to make their own replacement enzyme and has the potential to provide significant clinical and quality of life advantages over repeatedly administered enzyme replacement therapy, which is the current standard of care for MPS II and a number of other genetic diseases,” said Sandy Macrae, M.B., Ch.B., Ph.D., Sangamo’s president and chief executive officer. “Unlike conventional, non-integrating AAV gene therapy that has the potential to ‘wash out’ over time as the patient’s liver cells divide and turn over, SB-913 has the potential to provide a uniquely durable solution through genome editing. Ultimately, our target population will include pediatric patients who can benefit from a more durable solution. We believe that a ZFN-mediated genome editing approach can best serve the broadest group of MPS II patients and their families.”

Dr. Macrae added, “I am very impressed by the ability of our research and development team to successfully prepare and file several IND applications in short succession, while simultaneously working to open study sites and initiate two clinical trials for hemophilia B and MPS I. One of my first objectives as CEO will be to further expand our clinical team so that we continue to build on these achievements and swiftly translate our efforts into clinical data.” (Original Source)

Shares of Sangamo Biosciences are up over 10% to $6.20 in pre-market trading. SGMO has a 1-year high of $11.49 and a 1-year low of $4.63. The stock’s 50-day moving average is $6.36 and its 200-day moving average is $6.67.

On the ratings front, Sangamo has been the subject of a number of recent research reports. In a report issued on June 8, Jefferies Co. analyst Gena Wang reiterated a Buy rating on SGMO, with a price target of $14, which implies an upside of 149.1% from current levels. Separately, on May 3, Wells Fargo’s Jim Birchenough reiterated a Buy rating on the stock .

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Gena Wang and Jim Birchenough have a total average return of -20.8% and 17.7% respectively. Wang has a success rate of 10.5% and is ranked #3810 out of 3974 analysts, while Birchenough has a success rate of 42.5% and is ranked #109.

Sangamo BioSciences, Inc. is a clinical stage biopharmaceutical company focused on the research, development and commercialization of engineered DNA-binding proteins as novel therapeutics to treat unmet medical needs. It is engaged in developing zinc finger DNA-binding proteins (ZFPs) for therapeutic gene regulation and modification. Its zinc finger DNA-binding proteins (ZFPs) could be engineered to make ZFP nucleases (ZFNs) proteins, which could be used to modify DNA sequences in various ways for genome editing and gene regulation and ZFP transcription factors (ZFP TFs) proteins could be used to turn genes on or off. The company has ongoing Phase 2 clinical trials to evaluate the safety and efficacy of a novel ZFP Therapeutic for the treatment of HIV/AIDS. Its other therapeutic programs are focused on monogenic diseases, including hemophilia, Huntington’s disease and hemoglobinopathies such as beta-thalassemia and sickle cell anemia.