Sangamo Biosciences, Inc. (Public, NASDAQ:SGMO)

Discussion in 'Men's Economics' started by Michael Scally MD, Sep 15, 2011.

  1. Michael Scally MD

    Michael Scally MD Doctor of Medicine

    Sangamo Biosciences, Inc. (Public, NASDAQ:SGMO) Sangamo BioSciences
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    SEC: 0001001233 (see all company filings)

    Sangamo BioSciences, Inc. (Sangamo), incorporated on June 22, 1995, is engaged in the research, development and commercialization of zinc finger deoxyribonucleic acid (DNA)-binding proteins (ZFPs), a naturally occurring class of proteins, and develop a technology platform. ZFPs can be engineered to make ZFP transcription factors (ZFP TFs), proteins that can be used to turn genes on or off, and ZFP nucleases (ZFNs), proteins that enable it to modify DNA sequences in a range of ways. The Company focuses on the development of human therapeutics and it is building a pipeline of ZFP Therapeutics. Its ZFP Therapeutic, SB-509, a plasmid formulation of a ZFP TF activator of the vascular endothelial growth factor-A (VEGF-A) gene, is under evaluation in a phase-IIb clinical trial for moderately severe diabetic neuropathy (DN). The Company has an ongoing phase-I/II clinical trial and two phase-I trials to evaluate the first therapeutic application of its ZFN technology, SB-728-T, a ZFN-modified T-cell product for the treatment of human immunodeficiency virus (HIV)/ acquired immune deficiency syndrome (AIDS). It has preclinical development programs of ZFP Therapeutics in Parkinson’s disease, hemophilia B, neuropathic pain, and neuroregenerative programs in spinal cord injury, traumatic brain injury and stroke. In addition, it has research stage programs in monogenic diseases, genetic conditions, which result from a defect in a single gene, including hemophilia and other hemoglobinopathies, and certain immunodeficiencies.

    ZFP Therapeutic Product Development Programs

    The Company’s product candidates include SB-509, SB-728-T and SB-313xTZ. Sangamo is developing SB-509, a disease-modifying therapeutic approach, to address the underlying nerve damage characteristic of DN. SB-509 is an injectable formulation of plasmid DNA, which encodes a ZFP TF designed to up-regulate the patient’s own gene encoding VEGF-A. VEGF-A has been demonstrated to have both angiogenic and direct neuroproliferative, and neuroregenerative and neuroprotective properties. The VEGF-A gene encodes multiple forms (isoforms) of the VEGF-A protein, which exhibit slightly different properties and bind to different VEGF-A receptors. It has completed preclinical studies of VEGF-A activation using its ZFP Therapeutic, SB-509, in animal models of DN and demonstrated that single and repeat intramuscular injections of SB-509 in rats with diabetes resulted in protection of nerve function in the treated limb as measured by sensory and motor nerve conduction velocities. It is evaluating SB-509 in an ongoing double-blind, placebo-controlled phase-IIb clinical trial (SB-509-901) in subjects with moderately severe DN. While not envisioned as a cure for Amyotrophic Lateral Sclerosis (ALS), its therapeutic approach is designed to be disease-modifying, to protect and restore the function of damaged nerves in order to preserve muscle strength.

    The Company’s therapeutic approach aims to use its ZFN-mediated gene editing technology to replicate a naturally occurring human mutation. CCR5 is a co-receptor for HIV entry into T-cells and, if CCR5 is not expressed on their surface, HIV infects them with lower efficiency. It is using ZFN-mediated gene disruption technology to disrupt the CCR5 gene in cells of a patient’s immune system to make these cells permanently resistant to HIV infection. In October 2010, it also initiated a new phase I/II study (SB-728-T-1002) to evaluate SB-728-T in HIV-infected individuals who are not yet on highly active antiretroviral therapy (HAART). It also expanded its SB-728-T-901 study to include an additional cohort of subjects that are failing HAART. In collaboration with clinicians at City of Hope (COH), it is developing a ZFP Therapeutic, which uses its ZFN technology to disrupt the expression of the gene encoding the glucocorticoid receptor in T-cells. Its collaborators have developed an engineered protein known as an IL-13 zetakine that, when expressed in cytotoxic or killer T-cells, enables them to seek out and destroy glioblastoma cells in the brain.

    ZFP Therapeutic Pre-clinical Stage Programs

    In addition to its ongoing phase-IIb clinical trial in DN, and phase-I studies in HIV/AIDS and glioblastoma, the Company has multiple preclinical-stage programs, which is ZFP TF and ZFN molecules in animal efficacy studies. It has identified ZFP TF candidates that repress the expression of two of these pain targets, Trk-A and PN3, in cell-based models. Trk-A and PN3 fall into the class of non-druggable targets. It has incorporated these ZFP TFs into gene transfer vectors and have demonstrated a statistically significant reduction of pain in an animal model of bone cancer pain after treatment with Sangamo’s ZFP TF repressor of Trk-A. Further animal studies are ongoing. In collaboration with several academic labs, it is evaluating its ZFP TF activator of the VEGF-A gene in pre-clinical animal efficacy models of SCI, TBI and stroke. It has presented data, which demonstrate an effect on both recovery of hind-limb function and spinal cord tissue preservation following treatment at the time of injury with its ZFP TF activator of VEGF-A in a severe model of SCI. Further preclinical studies are ongoing to evaluate SB-509 in ischemia models of TBI and stroke and in SCI models to investigate dosing regimens. It also has several research stage ZFN-mediated gene modification programs in progress. These initiatives include programs in monogenic diseases, including hemoglobinopathies, such as sickle cell anemia, and immune system disorders, such as X-linked severe combined immunodeficiency (X-linked SCID).

    The Company competes with Eli Lilly and Company, Merck & Co., Inc., Takeda Pharmaceutical Company Limited, Pfizer, Inc., Exelixis Inc., Rigel Pharmaceuticals, Gilead, Amgen Inc., Genentech, Inc., Human Genome Sciences, Biogen Idec, Johnson & Johnson, Amsterdam Molecular Therapeutics, GenVec Inc., VIRxSYS Corporation, Dendreon, Alnylam Pharmaceuticals, Inc., Isis Pharmaceuticals, Inc., Regulus Therapeutics, LLC, Cellectis SA and Precision BioSciences, Inc.
  2. Michael Scally MD

    Michael Scally MD Doctor of Medicine

    Promising signs emerging from HIV gene therapy trials - September 12, 2011
    Nature News Blog: Promising signs emerging from HIV gene therapy trials