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Sangamo BioSciences Announces Publication in Nature Medicine Demonstrating Use of ZFN Technology to Engineer Safer and More Effective Cancer Treatments

Award-Winning Study Demonstrates Use of ZFN Technology to Develop Novel Therapeutics Designed to Kill Cancer Cells
RICHMOND, Calif., April 2, 2012 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced today the publication of a preclinical study demonstrating the use of zinc finger nucleases (ZFNs) to engineer safer and more potent cancer immunotherapies. The study, published in Nature Medicine, advances the development of novel, cell-based therapies for the treatment of a broad range of cancers. 

The studies were performed in the laboratory of Chiara Bonini, M.D., Head of the Experimental Hematology Unit, San Raffaele Hospital, Milan, in collaboration with Luigi Naldini, Head of TIGET, San Raffaele Hospital, and Sangamo scientists. Dr. Bonini also presented the data at the opening session of the 2012 Annual Meeting of the European Group for Blood and Marrow Transplantation (EBMT). The importance of the work has been recognized with the Van Bekkum Award, the most prestigious EBMT award for the best abstract submitted to the physician's program and is selected by the EBMT Board. The meeting is being held from April 1- 4, 2012, in Geneva, Switzerland.

"These data represent a significant advance in maximizing the potential safety and efficacy of adoptive T-cell therapies for cancer," said Dr. Bonini. "While we can reprogram cells of the immune system to preferentially recognize and kill tumors, several hurdles remain that limit the potential of this approach. ZFN-based genome editing enables us to specifically modify these redirected cells and disrupt genes that interfere with their potency and specificity, thus generating a safer, more efficacious therapeutic product."

Cancer immunotherapy uses the immune system, specifically CD8+ T-cells, that have been "redirected" to seek out and destroy tumors.  These redirected T-cells are genetically engineered to express new cell surface receptors that specifically recognize tumor cells. However, CD8+ T-cells have a natural specificity for different (non-tumor) targets and the resulting competition between the natural and the tumor-targeting gene products limits the potency of this cell therapy. More importantly, it can also make the cells "self-reactive," leading to graft versus host disease (GvHD).

In this study, ZFNs were used to specifically disrupt the native T-cell receptor (TCR) genes in these tumor-directed CD8+ T-cells resulting in an enhanced immunotherapeutic product with potent anti-cancer activity coupled with the elimination of GvHD in a mouse model. The data are described in a Nature Medicine paper entitled "Editing T-cell Specificity Towards Leukemia by Zinc Finger Nucleases and Lentiviral Gene Transfer" which appears as an Advance Online Publication on the Nature Medicine website .

"We congratulate our scientists and collaborators on this potentially significant medical advance in the treatment of cancer, and are gratified by the continued recognition of our ZFN-genome editing technology to generate both novel therapeutic approaches and expand the utility of existing therapies," stated Edward Lanphier, Sangamo's president and chief executive officer. "Our clinical and preclinical programs are currently focused on developing ZFP Therapeutics for HIV/AIDS and monogenic and rare diseases. However, as these data in oncology applications demonstrate, our ZFP technology platform enables the development of novel and innovative therapeutics to address a broad range of indications."

About Sangamo

Sangamo BioSciences, Inc. is focused on research and development of novel DNA-binding proteins for therapeutic gene regulation and genome editing. It has ongoing Phase 2 and Phase 1/2 clinical trials to evaluate the safety and efficacy of a novel ZFP Therapeutic® for the treatment of HIV/AIDS. Sangamo's other therapeutic programs are focused on monogenic diseases, including hemophilia and hemoglobinopathies such as sickle cell anemia and beta-thalassemia, and a program in Parkinson's disease. Sangamo's core competencies enable the engineering of a class of DNA-binding proteins known as zinc finger DNA-binding proteins (ZFPs). Engineering of ZFPs that recognize a specific DNA sequence enables the creation of sequence-specific ZFP Nucleases (ZFNs) for gene modification and ZFP transcription factors (ZFP TFs) that can control gene expression and, consequently, cell function. Sangamo has entered into a strategic collaboration with Shire to develop therapeutics for hemophilia and other monogenic diseases and has established strategic partnerships with companies in non-therapeutic applications of its technology including Dow AgroSciences and Sigma-Aldrich Corporation. For more information about Sangamo, visit the company's website at

ZFP Therapeutic® is a registered trademark of Sangamo BioSciences, Inc.

This press release may contain forward-looking statements based on Sangamo's current expectations. These forward-looking statements include, without limitation, the potential of ZFNs to advance cancer immunotherapy and treat other human diseases, research and development of novel ZFP TFs and ZFNs and therapeutic applications of Sangamo's ZFP technology platform. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties relating to the initiation and completion of stages of our clinical trials, whether the clinical trials will validate and support the tolerability and efficacy of ZFNs, technological challenges, Sangamo's ability to develop commercially viable products and technological developments by our competitors. For a more detailed discussion of these and other risks, please see Sangamo's SEC filings, including the risk factors described in its Annual Report on Form 10-K and its most recent Quarterly Report on Form 10-Q. Sangamo assumes no obligation to update the forward-looking information contained in this press release.

SOURCE Sangamo BioSciences, Inc.


Posted: April 2012