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Sangamo BioSciences Provides Update on Company's 2008 Progress and 2009 Objectives

NEW YORK, Dec. 3 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced that the company will provide an update on milestones achieved in 2008 and preview objectives for 2009 during its annual Investor and Analyst Briefing to be held in New York City today.

David Smoller, Ph.D., President of Sigma-Aldrich Corporation's Research Biotech Business Unit will discuss the progress and expected potential of the two companies' collaboration to commercialize zinc finger DNA-binding protein (ZFP) technology for research applications. Dr. Smoller will be joined by Edward Lanphier, Sangamo's president and CEO and other members of Sangamo's senior management team.

"Sigma is committed to providing breakthrough products to its customers in laboratories worldwide and we expect Sangamo's ZFP technology to be nothing short of revolutionary for research applications," commented Dr. Smoller. "Marketed under the trademark CompoZr, the zinc finger nuclease (ZFN) technology makes available, for the first time, simple, efficient genomic editing. CompoZr(TM) ZFN reagents can be used to efficiently create modified cell lines and model organisms with precise and heritable gene deletions, insertions or modifications using simple, widely used laboratory procedures. This is proven technology with strong potential."

"The past year has been a year of real progress for our ZFP technology platform," said Edward Lanphier, president and CEO of Sangamo. "We are successfully translating the broad applicability of our technology into opportunities for both novel human therapeutics and non-therapeutic industrial applications. Our unique hybrid business model maximizes the value of our ZFP technology platform and has enabled us to make significant progress in moving our lead ZFP Therapeutic into multiple Phase 2 clinical trials and advancing our preclinical pipeline, while maintaining a strong balance sheet. We have established important commercial agreements in the non-therapeutic aspects of our technology with Dow Agrosciences in plant agriculture and Sigma-Aldrich Corporation in research reagents. We also have agreements with Genentech and Pfizer in cell-line engineering. We plan to establish strategic partnerships around our therapeutic programs at points of value inflection and expect 2009 to be another year of significant accomplishments for Sangamo."

Mr. Lanphier continued, "We are pursuing a broad development program for our lead ZFP Therapeutic(TM), SB-509, an activator of vascular endothelial growth factor (VEGF) with three Phase 2 clinical trials ongoing for the treatment of diabetic neuropathy (DN) and one to evaluate its use in the treatment of amyotrophic lateral sclerosis (ALS). We have also advanced our preclinical pipeline and expect to file Investigational New Drug (IND) application for the first of our ZFN-based therapeutic programs for HIV/AIDS before the end of 2008.

"We are very pleased by the progress of our non-therapeutic collaborations this year. Our partner Sigma-Aldrich successfully launched the CompoZr brand of ZFN-based gene editing tools and we look forward to the increased interest and further validation of the technology that this broadening of the user-base will bring. We also transitioned ahead of schedule into the commercial phase of our collaboration with Dow AgroSciences (DAS) to apply our ZFP technology for plant agriculture and expect DAS to begin to sublicense the technology in that industry. The success of these activities is key to further enhancing the market-leading presence for our technology. We believe that our progress in advancing our technology platform, which is unique in its generation of novel, highly differentiated therapies and products, will create interest among potential pharmaceutical partners for our ZFP Therapeutic programs."

Sangamo Accomplishments in 2008

During the briefing several of the company's achievements will be highlighted including:


-- Presentation of the full data set from single dose Phase 1b study (SB-509-401) of SB-509 in subjects with diabetic neuropathy (DN) demonstrating that the drug is well-tolerated and statistically and clinically significant improvements in measures of nerve health at 180 days post treatment.

-- Initiation of repeat dosing Phase 2 clinical trials of SB-509 in stem cell mobilization (SB-509-703) and ALS (SB-509-801).

-- Completion of enrollment and expansion of Phase 2 clinical trial in subjects with moderate to severe DN (SB-509-701). Presentation of encouraging positive interim data from this trial.

-- Presentation of top line Phase 2 data from repeat dosing study of SB-509 in mild to moderate DN (SB-509-601) demonstrating that the drug is well-tolerated in a repeat-dosing setting; however, no significant differences were observed in improvement in measures of nerve health between placebo and treated subjects at primary evaluation point 180 days post treatment. Analysis of these data is ongoing.


-- Dow AgroSciences announces early exercise of an option for a commercial license for ZFP technology in plant agriculture.

-- Official launch of Sigma-Aldrich CompoZr platform of zinc finger nuclease (ZFN) reagents for gene-editing.

-- Entry into Research and License Agreement with Genentech, Inc. for cell line engineering and License Agreement with Open Monoclonal Technologies (OMT) and Roche for transgenic animal generation.


-- Sangamo updated guidance on its expectations of 2008 year end cash to $60M in cash and cash equivalents, which the company believes is sufficient to fund operations through 2010 based on current expense projections.

Preclinical / Research

-- Publication of data supporting Sangamo's first ZFN Therapeutic program for the treatment of HIV/AIDS in high-impact journal Nature Biotechnology demonstrating highly efficient and specific ZFN-modification of CCR5 in human T-cells resulting in an increase in CD4 T-cell counts and a reduction in viral load in an animal model of HIV infection (Perez et al., 2008, Nat Biotechnol.,26: 808).

-- Nature Biotechnology publication establishing the utility of ZFNs in the generation of transgenic animals and the potential broadening of the production of knockout transgenic models beyond mouse models (Doyon et al., 2008, Nat. Biotechnol. 26: 702).

-- Publication of data in the journal Proceedings of the National Academy of Sciences (PNAS) establishing Sangamo's ZFN technology as a new approach for rapid generation of "knock-out" cell lines (Santiago et al., 2008, PNAS 105: 5809).

Select 2009 Objectives

During the briefing Sangamo will also discuss the following objectives for 2009:

-- Sangamo expects to file an IND for SB-313 to evaluate the drug in a Phase 1 trial in subjects with glioblastoma.

-- The company expects to present data from its three ongoing Phase 2 clinical trials of SB-509 in subjects with DN, SB-509-601 in subjects with mild to moderate DN, SB-509-701 subjects with moderate to severe DN with a blocked nerve and SB-509-703 in stem cell mobilization.

-- Establishment of a strategic partnership for SB-509.

-- Sangamo estimates that it expects to end 2009 with approximately $40M in cash and cash equivalents, based on current expense projections and expected progress in existing corporate relationships.

The presentation from today's Investor and Analyst Briefing will be archived on Sangamo's website and is available via a link in the Investor section at under "Events and Presentations".

About Sangamo

Sangamo BioSciences, Inc. is focused on the research and development of novel DNA-binding proteins for therapeutic gene regulation and modification. The most advanced ZFP Therapeutic(TM) development program is currently in Phase 2 clinical trials for evaluation of safety and clinical effect in patients with diabetic neuropathy and ALS. Other therapeutic development programs are focused on cancer, HIV/AIDS, neuropathic pain, nerve regeneration, Parkinson's disease and monogenic diseases. Sangamo's core competencies enable the engineering of a class of DNA-binding proteins known as zinc finger DNA-binding proteins (ZFPs). By engineering ZFPs that recognize a specific DNA sequence Sangamo has created ZFP transcription factors (ZFP TF(TM)) that can control gene expression and, consequently, cell function. Sangamo is also developing sequence-specific ZFP Nucleases (ZFN(TM)) for gene modification. Sangamo has established strategic partnerships with companies outside of the human therapeutic space including Dow AgroSciences, Sigma-Aldrich Corporation and several companies applying its ZFP technology to enhance the production of protein pharmaceuticals. For more information about Sangamo, visit the company's web site at

This press release may contain forward-looking statements based on Sangamo's current expectations. These forward-looking statements include, without limitation, references to the research and development of novel ZFP TFs and ZFNs as ZFP Therapeutics, applications of Sangamo's ZFP TF technology platform to specific human disease as well as plant agriculture, high value research reagents and cell-line engineering, clinical trials of ZFP Therapeutics and expected filing of INDs, establishing strategic partnerships for SB-509 and other therapeutic programs, anticipated amount of cash and cash equivalents and sufficiency of cash reserve to fund operations. Actual results may differ materially from these forward-looking statements due to a number of factors, including technological challenges, uncertainties relating to the initiation, completion and outcome of stages of ZFP Therapeutic clinical trials, Sangamo's ability to develop commercially viable products and technological developments by our competitors. See Sangamo's SEC filings, and in particular, the risk factors described in Sangamo's Annual Report on Form 10-K and its most recent Quarterly Reports on Form 10-Q. Sangamo BioSciences, Inc. assumes no obligation to update the forward-looking information contained in this press release.

SOURCE Sangamo BioSciences, Inc.
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