Sangamo Biosciences, Inc. (NASDAQ:SGMO), the leader in therapeutic genome editing, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to SB-FIX, the company’s zinc finger nuclease (ZFN)-mediated genome editing product candidate for the treatment of hemophilia B. Sangamo expects to initiate a Phase 1/2 clinical study (SB-FIX-1501) in adult subjects with the disease in 2016.

“We are pleased with the FDA’s decision to grant orphan drug designation to SB-FIX for hemophilia B,” said Geoff Nichol, M.B., Ch.B., Sangamo’s executive vice president of research and development. “By enabling targeted integration of a therapeutic factor 9 gene, our ZFN-mediated genome editing approach may offer hemophilia B patients a therapeutic option that has potential advantages over conventional gene therapy approaches. We will enroll adult hemophilia patients into our first clinical trial, however, our goal is to move into pediatric patients, a population we believe could particularly benefit from a treatment that has the potential to provide lifelong expression of therapeutic levels of Factor IX protein.”

“Our mission is to translate our ground-breaking science into genetic therapies that transform patients’ lives,” said Sandy Macrae, M.B., Ch.B., Ph.D., Sangamo’s president and chief executive officer. “This is another positive step forward for the first therapeutic in vivo genome editing application cleared for clinical evaluation in humans. I am pleased with the progress that we have made to advance this program and we look forward to initiating a Phase 1/2 clinical trial by the end of 2016.”

The FDA grants orphan drug designation to investigational drugs and biologics that are intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. Orphan drug status is intended to facilitate drug development for rare diseases and provides several benefits to drug developers, including assistance with clinical study design and drug development, tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.

Sangamo’s In Vivo Genome Editing Approach

SB-FIX is designed as a single treatment strategy intended to provide stable, continuous production of Factor IX clotting protein (FIX) for the lifetime of the patient. Sangamo’s ZFN-mediated in vivo genome editing approach makes use of the albumin gene locus, a highly expressing and liver-specific genomic “safe-harbor site,” that can be edited with ZFNs to accept and express therapeutic genes. The approach is designed to enable the patient’s liver to permanently produce circulating therapeutic levels of a corrective protein product. The ability to permanently integrate the therapeutic gene in a highly specific targeted fashion significantly differentiates Sangamo’s in vivo genome editing approach from conventional AAV cDNA gene therapy approaches, which are non-integrating, and may “wash out” of the liver as cells divide and turn over. Ultimately, the target population for these programs will include pediatric patients and it will be important in this population to be able to produce stable levels of therapeutic protein for the lifetime of the patient. With such a large capacity for protein production (approximately 15g/day of albumin), targeting and co-opting only a very small percentage of the albumin gene’s capacity is sufficient to produce the needed replacement protein at therapeutically relevant levels with no significant effect on albumin production. (Original Source)

Shares of Sangamo closed last Friday at $4.32, down $0.01 or -0.23%. SGMO has a 1-year high of $9.91 and a 1-year low of $4.08. The stock’s 50-day moving average is $5.06 and its 200-day moving average is $5.86.

On the ratings front, Sangamo has been the subject of a number of recent research reports. In a report issued on August 19, Jefferies analyst Gena Wang reiterated a Buy rating on SGMO. Separately, on August 8, Cowen’s Ritu Baral 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 Ritu Baral have a total average return of -13.8% and 3.8% respectively. Wang has a success rate of 31% and is ranked #3965 out of 4147 analysts, while Baral has a success rate of 42% and is ranked #737.

The street is mostly Bullish on SGMO stock. Out of 4 analysts who cover the stock, 3 suggest a Buy rating and one recommends to Hold the stock.

Sangamo BioSciences, Inc. is a clinical stage biopharmaceutical company, which is focused on the research, development and commercialization of engineered DNA-binding proteins for therapeutic genome editing and gene regulation technology platform is enabled by the engineering of a class of transcription factors known as zinc finger DNA-binding proteins.