Sangamo Biosciences, Inc. (NASDAQ:SGMO), a leader in therapeutic genome editing, announced today the publication in Nature Biotechnology of data demonstrating efficient zinc finger nuclease (ZFN)-mediated, targeted gene insertion in human hematopoietic stem and progenitor cells (HSPCs). In addition, data published earlier this month in Nucleic Acids Research, demonstrate that similar efficiencies can be obtained in primary human T-cells. Sangamo’s genome editing method provides a precisely targeted and therefore potentially safer approach than traditional randomly integrative gene therapies, such as lentiviral vectors, for the treatment of human diseases of the blood and immune system, and the generation of T-cell based cancer immunotherapies.
“These studies present new and substantial data affirming that Sangamo’s ZFN genome-editing platform provides precise, robust targeted insertion of therapeutic genes, in contrast to conventional integrating vector approaches, such as lentiviral vectors that insert genes randomly,” stated Edward Lanphier, Sangamo’s president, and chief executive officer. “Our extensive clinical experience and a substantial body of research, documents the accuracy and efficiency of our ZFN technology, and positions it as a more versatile and potentially safer therapeutic strategy than other gene therapy and genome editing approaches. Our technology also provides an alternative manufacturing platform for cell therapies.”
Researchers capitalized on the fact that ZFN-mediated genome editing requires only transient expression of the ZFNs to effect a permanent change in the genome. They used messenger RNA and electroporation to deliver the ZFNs and a non-integrating vector, an adeno-associated virus (AAV) serotype 6, to provide a therapeutic DNA sequence, a so-called “donor template”, to HSPCs and CD4 and CD8 T-cells. The “donor template” can encode an entire gene that integrates at the targeted site of ZFN action. This delivery approach achieved efficiencies of gene transfer and targeted integration of the therapeutic DNA sequence, particularly in more primitive longer-lasting stem cells and T-cells, to levels that can be used therapeutically to potentially treat a range of monogenic diseases.
“Efficient targeted integration of a therapeutic gene into both hematopoietic stem cells and T-cells represents a major step forward in the quest for more precise and safer gene-based therapies,” stated Michael Holmes, Ph.D., Sangamo’s vice president, research, and a corresponding author on both papers. “Importantly, in our hematopoietic stem cell work with our scientific collaborators at USC, we successfully modified a class of the most pluripotent stem cells, leading to long-term production of ZFN-modified cells across all blood cell lineages following transplantation into immune deficient mice. Our results in these two studies provide a robust strategy for additional therapeutic applications of ZFN-mediated genome editing in HSPCs and T-cells.” (Original Source)
Shares of Sangamo Biosciences opened today at $7.99 and are currently trading down at $7.94. SGMO has a 1-year high of $19.25 and a 1-year low of $5.30. The stock’s 50-day moving average is $6.57 and its 200-day moving average is $9.30.
On the ratings front, Sangamo Biosciences has been the subject of a number of recent research reports. In a report issued on September 28, Piper Jaffray analyst Charles Duncan reiterated a Buy rating on SGMO, with a price target of $17, which implies an upside of 112.8% from current levels. Separately, on September 4, Cowen’s Ritu Baral reiterated a Buy rating on the stock .
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Charles Duncan and Ritu Baral have a total average return of 12.4% and 18.9% respectively. Duncan has a success rate of 47.9% and is ranked #450 out of 3829 analysts, while Baral has a success rate of 47.9% and is ranked #88.
The street is mostly Bullish on SGMO stock. Out of 4 analysts who cover the stock, 4 suggest a Buy rating . The 12-month average price target assigned to the stock is $23.50, which implies an upside of 194.1% from current levels.
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.