Advertisement covers The 19th Conference on Retroviruses and Opportunistic Infections (CROI 2012)

Press Release

Sangamo Presents New Clinical Data at CROI 2012 Demonstrating Persistent Positive Effects of ZFN Therapeutic for "Functional Cure" of HIV/AIDS

Data Highlight Positive Effects of SB-728-T on Immune System and Viral Load Reduction and Further Validate Strategy of Ongoing Phase 2 Trials

March 8, 2012

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Abstract #433 "A Single Infusion of Zinc Finger Nuclease CCR5 Modified Autologous CD4 T Cells (SB-728-T) Increases CD4 Counts and Leads to Decrease in HIV Proviral Load in an Aviremic HIV-infected Subject" Wednesday, March 7, 2012

In one INR subject (01-104) who received a single dose of SB-728-T (10 billion cells) an inflammatory event unrelated to SB-728-T infusion was observed in the gut mucosa by histology and surface marker staining of mucosal inflammatory cells from 3 to 6 months after infusion. Subsequently, a greater than 1 log increase in SB-728-T was observed locally in the gut, preceding resolution of the inflammation.

This study demonstrated:

  • That SB-728-T is functionally active and capable of participating in an immunologic response and has the potential to maintain or restore health in the gastrointestinal tract.
  • An associated increase in total CD4+ T-cells and percent of ZFN-CCR5-modified CD4+ T-cells in the gut mucosa in response to the inflammation event.
  • Analysis of individual T-cell clones confirmed that this significant increase in SB-728 was specific to the gut mucosa and indicative of a precise response by these cells to local gut inflammation.
  • HIV-DNA levels in both the gut mucosa and the periphery decreased from peak levels by about 1 log, coinciding with the timeline of significant increases in SB-728-T at the site of inflammation suggesting a possible SB-728-T antiviral effect.

"The data presented at CROI further support our fundamental rationale for SB-728-T and, importantly, the design of our ongoing Phase 2 clinical trials which will enable us to quickly maximize the impact of SB-728-T on viral load and the overall immune system of HIV-infected individuals," said Geoff Nichol, M.B., Ch.B., Sangamo's executive vice president of research and development. "Our ZFP technology provides a novel platform for the creation of transformational ZFP Therapeutics and we are focused on using it to develop curative therapies for HIV and multiple monogenic and rare diseases."

Webcasts of all the presentations at CROI 2012 can be accessed via the following link

About SB-728-T

SB-728-T is an autologous CD4 T-cell product in which the gene for CCR5, a co-receptor for HIV entry, is modified via ZFN-mediated genome editing to disrupt the CCR5 protein. T-cells with a disrupted CCR5 protein are resistant to infection by the most common strain of HIV.

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, 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, references relating to research and development of novel ZFP TFs and ZFNs and therapeutic applications of Sangamo's ZFP technology platform for the treatment of HIV/AIDS, including a potential functional cure of HIV/AIDS, the expansion of clinical studies of SB-728-T in HIV-infected individuals and the initiation of additional preclinical and clinical studies of ZFN-gene modification. 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 BioSciences, Inc. assumes no obligation to update the forward-looking information contained in this press release.

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This article was provided by Sangamo BioSciences, Inc. .

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