Sangamo BioSciences, Inc. Provides Comprehensive Overview of Company's ZFP Therapeutic&#0174 Development Programs at Its 2012 Analyst Briefing

Published: Dec 07, 2012

NEW YORK, Dec. 6, 2012 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO), announced that the company is providing an update on its technology platform advancements and pipeline of ZFP Therapeutics® as well as near- and mid-term operating goals during an Analyst and Investor Briefing being held today in New York City. The presentation, by members of Sangamo management team, will be webcast live beginning at 5:00 pm ET and can be accessed via a link on the Company's website.

"We are very excited to share the progress that we have made in advancing our zinc finger DNA binding protein (ZFP) technology and ZFP Therapeutic programs, particularly in the area of monogenic diseases where we have developed a new, highly disruptive therapeutic platform for protein replacement therapies," said Edward Lanphier, president and CEO of Sangamo. "This approach can be leveraged across multiple diseases with the goal of engineering genetic cures."

"Sangamo's proprietary gene-editing and gene-regulation technology is unique in its ability to generate novel, highly differentiated therapies that act at the DNA-level," stated Geoff Nichol, M.B., Ch.B., Sangamo's executive vice president of research and development. "This capability enables us to address the source of a wide variety of genetic diseases and potentially cure them. In addition to two ongoing Phase 2 clinical trials of SB-728-T, which may afford a functional cure for HIV/AIDS, we have a rich pipeline of preclinical programs. These include ZFP Therapeutics for hemophilia and Huntington's disease, which we are developing in collaboration with Shire, as well as Sangamo's programs in hemoglobinopathies (sickle cell disease and beta-thalassemia) and lysosomal storage diseases (LSDs). Based upon our ZFP Therapeutic approach and the disease targets that we have selected, we expect to file up to seven new INDs by 2015."

"Commercially, our business model has enabled us to monetize the value of our ZFP technology platform and maintain a strong balance sheet through partnerships with Dow AgroSciences in plant agriculture and Sigma-Aldrich Corporation in life science research reagents, while making significant progress in advancing our ZFP Therapeutics programs," continued Mr. Lanphier. "Beyond our very significant collaboration with Shire, we plan to establish additional high-value strategic partnerships around selected therapeutic programs at points of realizable value inflection while continuing to advance proprietary programs and increasingly forward integrate."

Program Highlights
During the briefing members of Sangamo's management team will discuss recent achievements and objectives for the 2013-2015 time period including:


  • Sangamo plans to present preliminary data in the first half of 2013 from its clinical trials of SB-728-T (SB-728-1101 and SB-728-902 Cohort 5) for the treatment of HIV/AIDS and expects to have the full data set from these trials by the end of 2013. Both studies are designed to maximize the engraftment of T-cells in which both copies of the CCR5 gene have been disrupted making the cells resistant to infection by HIV. This creates a reservoir of cells that cannot be infected by the virus but are available to mount an immune response with the goal of providing a "functional cure" for HIV.

Preclinical / Research

  • Sangamo will outline its strategy to enable the potential filing of seven Investigational New Drug (IND) Applications by the end of 2015.
  • The Company will introduce a new, highly disruptive "In Vivo Protein Replacement Platform" driven by Sangamo's ZFP nuclease (ZFN) technology which is potentially curative and leverageable across many monogenic diseases, such as hemophilia and LSDs (e.g. Gaucher, Fabry, and Pompe disease) that are currently treated by regular infusions of enzyme replacement therapy (ERT). Sangamo's novel approach uses a naturally highly expressed gene to drive continuous production of active forms of these proteins from the liver such that they are present at stable levels in the bloodstream thus reducing or eliminating the need for ERT.
  • The Company will update investors on recent preclinical progress in ZFP Therapeutics programs with the goal of engineering a genetic cure for hemophilia, Huntington's disease and hemoglobinopathies (sickle cell disease and beta-thalassemia) and outline the key next steps and timelines to IND filing.


  • Sangamo will provide a near- and mid-term financial overview including the anticipated amount of cash and cash equivalent of $40-$45 million by the end of 2015 assuming completion of milestones associated with existing partnerships but not including any additional funding from new partnerships, research grants or equity financing transactions.

Sangamo's 2012 Analyst and Investor Briefing is being webcast live and will be available on Sangamo's website at 5:00pm ET via a link in the Investor section at under "Events and Presentations". A replay of the webcast will be archived and available on the website approximately two hours after the presentation.

About Sangamo
Sangamo BioSciences, Inc. is focused on research and development of novel DNA-binding proteins for therapeutic gene regulation and genome editing. 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. Sangamo's other therapeutic programs are focused on monogenic diseases, including hemophilia, Huntington's disease and hemoglobinopathies such as sickle cell anemia and beta-thalassemia. 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 AG to develop therapeutics for hemophilia, Huntington's disease 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, 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, cures using ZFP Therapeutics, clinical trials of ZFP Therapeutics and expected filing of INDs, establishing strategic partnerships for SB-728-T and other therapeutic programs, achievement of milestones under existing partnerships, 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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