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Sangamo Biosciences Inc. Message Board

  • Rob_Cos Rob_Cos Sep 14, 2007 1:34 PM Flag

    Talked to SGMO IR re: Tues presentation & Dow/Monsanto Deal

    Talked to SGMO Inv Relations about today's Dow/Monsanto corn deal and the HIV resistance T cell update on Tuesday....

    While the Dow/Monsanto deal appears to be based upon more traditional older technology she says its good news as it will be commercialized by 09 and then Dow will be able to really differentiate itself with the SGMO technology. She said there was a call on the Dow/Monsanto deal that did give SGMO some positive prominient mention - especially on the last analyst question which was asked by an analysts that also covers SGMO. I have found this PDF powerpoint that describes the deal but can't find the link to the call yet. I have asked her to send it to me. I will also be getting the full transcript of the call and will post it including the SGMO related points.

    Also great news - she told me that we will get some NEW updated info at this presentation on Tuesday:

    Sept 18th - 47th Interscience Conference on Antimicrobial Agents and Chemotherapy (ICAAC), which will be held in Chicago, Illinois, September 17 � 20, 2007. I have confirmed abstract 627 is SGMO related - HIV resistant T Cell abstract is important and about to enter Phase I (see related PR below)

    627 Holmes Establishment of HIV Resistant CD4 T Cells by Engineered Zinc Finger Protein Nucleases

    Sangamo BioSciences Presents First ZFP Therapeutic Data Demonstrating in Vivo Protection Against HIV
    Preclinical animal model shows ZFN-modified T-cells engraft and are protected from HIV infection

    RICHMOND, Calif., June 6, 2007 /PRNewswire-FirstCall via COMTEX News Network/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced the presentation of data from its program to develop a zinc finger DNA-binding protein (ZFP) Therapeutic(TM) for HIV/AIDS that demonstrate that ZFN-modified human T-cells are protected from HIV infection and have a selective survival advantage in a mouse model of HIV challenge. In the second half of 2007, Sangamo expects to initiate a Phase 1 clinical trial to test this HIV ZFP Therapeutic, working with Dr. Carl June, Director of Translational Research at the Abramson Family Cancer Research Institute and collaborators at the University of Pennsylvania.

    Sangamo's ZFP nuclease (ZFN(TM)) technology can be used to make human primary T-cells resistant to HIV infection by permanently modifying the DNA sequence encoding CCR5, an essential co-receptor for the entry of HIV into immune cells. Individuals have been identified who carry a natural mutation of the CCR5 gene, CCR5-delta32, which confers resistance to HIV infection. HIV infection kills or impairs cells of the immune system, progressively destroying the body's ability to fight infections resulting in AIDS (Acquired Immune Deficiency Syndrome). Individuals diagnosed with AIDS are susceptible to life-threatening diseases or opportunistic infections, which are caused by microbes that usually do not cause illness in people with healthy immune systems. Sangamo's initial approach is to modify the CCR5 gene in T-cells to provide patients with a reservoir of ZFN-modified HIV-resistant immune cells that can fight opportunistic infections and the virus itself.

    Continued in first reply to this Topic....

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    • In addition, Sangamo sponsored a Symposium chaired by Donald Kohn, M.D., Director, Gene, Immune and Stem Cell Therapy Program at Children's Hospital Los Angeles, which featured the following presentations:

      -- ZFPs as Therapeutics - Applications and Advantages of the Technology
      -- Philip Gregory, D. Phil., Vice President, Research, Sangamo
      BioSciences, Inc.
      -- Therapeutic Applications of a ZFP TF activator of VEGF-A
      -- Dale Ando, M.D., CMO and Vice President, Therapeutic Development,
      Sangamo BioSciences, Inc.
      -- ZFN Mediated Disruption of CCR5 to Create CCR5 deficient CD4 Cells for
      HIV Therapy
      -- Carl June, M.D., Director, Translational Research, Abramson Family
      Cancer Research Institute, University of Pennsylvania School of
      -- Gene Editing & Site-Specific Gene Addition in Human Stem Cells Using
      ZFN and Integrase-Defective Lentiviral Vectors
      -- Luigi Naldini, M.D., Ph.D., Professor of Cell and Tissue Biology,
      School of Medicine, Scientific Co director, The San Raffaele
      Telethon Institute for Gene Therapy (HSR-TIGET)
      -- Engineering Cellular Scalpels for Excising Invasive Tumor Cells of
      Malignant Glioma
      -- Michael Jensen, M.D., Associate Chairman, Division of Cancer
      Immunotherapeutics and Tumor Immunology, Beckman Research Institute,
      City of Hope

      A replay of the Symposium is available on Sangamo's website in the Investor section under Events and Presentations and can be accessed via the following link

    • "Scientists from Sangamo Biosciences approached me with an extraordinary proposition," stated Dr. June. "They told me that they had the means to create CD4+ T-cells with permanent modification of the CCR5 gene and a robust resistance to HIV infection. This exciting collaboration, involving my colleagues at the University of Pennsylvania and the team of scientists at Sangamo has rapidly progressed from that idea to data demonstrating that ZFN modified cells are HIV resistant and have a selective survival advantage when reintroduced into an animal. This is consistent with our aim of reconstituting a functional HIV-resistant immune system in patients infected with the virus.

      "This is the first presentation of in vivo data from our program to develop a ZFP Therapeutic for the treatment of HIV, and it is significant that these animal data confirm our earlier observations in isolated cells," stated Dale Ando, M.D., Sangamo's vice president of therapeutic development and chief medical officer. "Previously, we had shown that ZFN-modified primary human T-cells are resistant to HIV infection not only surviving continuous exposure to HIV but selectively expanding in cell culture to the point that they represented the vast majority of cells in the population at the end of the experiment. In these more recent data we show that that observation holds true when these HIV-resistant cells are tested in a mouse model of HIV infection and demonstrate that after ZFN-modification the cells are stable and have a selective advantage in the presence of HIV."
      In the experiments presented by Dr. June and Sangamo scientist, Michael Holmes, Ph.D., isolated human T-cells were treated with ZFNs specific for CCR5. The modified cells were infused into a mouse model, the NOG mouse, with or without a source of HIV. The NOG mouse lacks an immune system and thus does not reject the human cells. After 33 days, the mice were sacrificed and their blood analyzed for the presence of ZFN-modified cells. Researchers determined that ZFN-modified cells engrafted normally in the mouse and that the proportion of modified cells present at the end of the experiment was statistically significantly higher in mice in the presence of HIV infection (p=0.008). These data suggest that, in the presence of HIV, the ZFN-modified cells have a selective advantage and evade HIV infection and destruction.

      "These animal data are very encouraging as we prepare to initiate a Phase 1 clinical trial in HIV/AIDS with this ZFP Therapeutic in the second half of 2007," stated Edward Lanphier, Sangamo's president and CEO. "By administering ZFNs to patients' T-cells, the goal is to provide HIV-infected individuals with a reservoir of healthy and uninfectable immune cells that would be available to combat opportunistic infections and HIV itself. We believe that using ZFNs to permanently modify the CCR5 gene in T-cells and thus directly block HIV from entering cells may have a number of advantages over the systemic effects of CCR5 antagonists in development."

      The data were presented on June 1, 2007 at the 10th Annual Meeting of the American Society of Gene Therapy (ASGT), which was held in Seattle, Washington. In addition to Dr. Holmes' presentation, which was selected for the prestigious Presidential Symposium, Sangamo scientists and collaborators presented data in six oral presentations and three posters describing preclinical data from Sangamo's ZFP Therapeutic programs. These included programs in pain, nerve crush and spinal cord injury, macular degeneration and glioblastoma.

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