Sangamo BioSciences and Collaborators Present Data Showcasing Broad Applications of ZFP Therapeutics®

Monday, May 23, 2011 General News
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Twenty Presentations Highlight Programs in HIV/AIDS, Monogenic Diseases, Neuroregeneration and Stem Cell Applications

RICHMOND, Calif., May 23, 2011 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced today

that data from clinical, preclinical and research-stage programs focused on the development of zinc finger DNA-binding protein (ZFP) Therapeutics® were described in twenty presentations given by Sangamo scientists and collaborators at the 14th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT).  The meeting was held in Seattle from May 18-21, 2011.

"Sangamo's ZFP technology is revolutionizing cell and gene therapies," stated Barrie Carter, Ph.D., a member of Sangamo's scientific advisory board and President of ASGCT for 2010-2011. "ZFP Nuclease (ZFN) technology provides an efficient and precise process for editing any DNA sequence in a cell, making development of therapeutic gene-editing applications a reality.  Sangamo has ongoing human clinical trials to evaluate ZFN-based therapeutics in HIV/AIDS and ZFP transcription factors (ZFP TFs) in neuroregeneration. As this technology functions at the DNA level, it can be applied to numerous diseases and any disease-related gene."

ASGCT meeting presentations included previously described preliminary clinical data from Sangamo's ongoing Phase 1 clinical trial in HIV/AIDS (SB-728-902) as well as data from preclinical and research-stage human therapeutic programs.  Therapeutic areas included ZFN-based approaches to infectious diseases such as HIV/AIDS and monogenic diseases such as hemophilia, ADA-SCID, retinal neurodegeneration and epidermolysis bullosa.  Positive preclinical data were also presented from studies of Sangamo's ZFP TF activator of the vascular endothelial growth factor-A (VEGF-A) gene in an animal model of stroke.

"These data presentations illustrate the broad range of potential applications for ZFP Therapeutics including monogenic diseases, infectious diseases and neuroregeneration, as well as new approaches to tissue engineering and stem cell modifications," said Edward Lanphier, Sangamo's president and CEO. "Using our ZFP technology we can modify and regulate genes with singular specificity and high efficiency. Importantly, we can directly modify genes, correcting inherited mutations and restoring function while preserving the gene's natural regulation. These presentations demonstrate the rapid progress that Sangamo scientists and our collaborators have made in the development of our ZFP technology platform for the generation of novel therapeutic approaches for the treatment of unmet medical needs."

ASGCT Highlights

  • Sangamo scientist, Michael Holmes, Ph.D.,was a selected speaker at this year's Presidential Symposium and gave a presentation entitled "In vivo Delivery of Zinc Finger Nucleases Mediates Genome Editing to Correct Genetic Disease." (Abst# 49). Additional presentations covered ZFN-mediated gene editing approaches for monogenic diseases (Abst# 26, 93, 268, 423, 498, 631, 633, 648 and 689).
  • Sangamo's collaborator, Paula Cannon, Ph.D., Associate Professor of Molecular Microbiology & Immunology at the Keck School of Medicine of the University of Southern California was invited to chair the session, "Emerging Field Review: HIV Gene Therapy," in which Carl June, M.D., Director of Translational Research at the Abramson Family Cancer Research Institute at the University of Pennsylvania School of Medicine also presented.
  • Several presentations highlighted Sangamo's extensive HIV program, including clinical data from Sangamo's SB-728-T program (Abst# 598), preclinical data from Sangamo's program to disrupt the CCR5 gene in hematopoietic stem cells (Abst# 314 and 99) and positive preclinical data demonstrating ZFN-mediated disruption of  another co-receptor for HIV infection, CXCR4 (Abst# 484).

All abstracts for the meeting are available online at http://www.asgct.org/am11/index.php.

About Sangamo

Sangamo BioSciences, Inc. is focused on research and development of novel DNA-binding proteins for therapeutic gene regulation and modification.  The most advanced ZFP Therapeutic® development program is currently in a Phase 2b clinical trial for evaluation of safety and clinical effect in patients with diabetic neuropathy.  Sangamo also has a Phase 1 / 2 clinical trial and two ongoing Phase 1 clinical trials to evaluate safety and clinical effect of a treatment for HIV/AIDS as well as a Phase 1 trial of a treatment for recurrent glioblastoma multiforme. Other therapeutic development programs are focused on Parkinson's disease, monogenic diseases, neuropathic pain and nerve regeneration.  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 TFs) that can control gene expression and, consequently, cell function.  Sangamo is also developing sequence-specific ZFP Nucleases (ZFNs) for gene modification.  Sangamo 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 www.sangamo.com.

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 research and development of novel ZFP TFs and ZFNs as ZFP Therapeutics and therapeutic applications of Sangamo's ZFP technology platform to specific human diseases and unmet medical needs, including monogenic diseases, neuroregeneration and stem cell applications. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties relating to whether clinical trials will validate and support tolerability and efficacy of ZFP Therapeutic approaches, technological challenges, 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 the Company's Annual Report on Form 10-K and 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.



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