Sangamo BioSciences ZFP Therapeutic Program in HIV/AIDS Featured at Three Major Scientific Conferences in October 2014

RICHMOND, Calif., Oct. 16, 2014 /PRNewswire/ -- Sangamo BioSciences, Inc. (NASDAQ: SGMO) announced that Company scientists and clinicians, as well as academic collaborators, were invited speakers at three major scientific meetings in Europe and the United States. Their presentations covered a number of therapeutic uses of Sangamo's novel zinc finger protein (ZFP) technology, but were primarily focused on reviewing the progress of the Company's ZFP Therapeutic^® program in HIV/AIDS.  Presentations included a review of current clinical data with SB-728-T as well as the design of Sangamo's ongoing Phase 2 clinical trial (SB-728-mR-1401) and the preclinical rationale for targeting CCR5 in hematopoietic stem cells, which is expected to enter clinical testing in late 2014.

"Participation in these key scientific forums—the NIH 'Strategies for an HIV Cure 2014' meeting, the European Society of Gene and Cell Therapy (ESGCT) Annual Meeting, and most recently, the British HIV Association (BHIVA) Autumn Conference—underscores the tremendous interest from the international scientific and clinical communities in Sangamo's progress toward developing a ZFP Therapeutic that may replicate natural, durable resistance to HIV infection," said Edward Lanphier, Sangamo's president and CEO. "Sangamo's ongoing Phase 2 trial incorporates all that we have learned from previous trials about the potential mechanism of this novel therapeutic clinical approach, and we believe the data produced will provide a clear path to pivotal studies. We expect to accrue all subjects onto the clinical trial by the end of 2014 and to present data in 2015."

This week, October 15-17, at the "Strategies for an HIV Cure 2014" conference organized by the National Institute for Allergies and Infectious Diseases at the NIH, Philip Gregory, D.Phil., Sangamo's senior vice president, research, and CSO, will present an overview of the SB-728-T program, along with Sangamo collaborators Paula Cannon, Ph.D., Associate Professor Molecular Microbiology & Immunology, Pediatrics, Biochemistry & Molecular Biology, Keck School of Medicine, University of Southern California; Pablo Tebas, M.D., Professor of Medicine at the Hospital of the University of Pennsylvania; and Hans-Peter Kiem, M.D., Jose Carreras/E. Donnall Thomas Endowed Chair for Cancer Research at the Fred Hutchinson Cancer Research Center. 

Dr. Gregory will also be presenting an overview of the SB-728-T clinical program at a "bench to bedside" discussion forum of the annual ESGCT meeting, October 23-26, which will be held in The Hague. Dr. Gregory and Sangamo collaborator Luigi Naldini, M.D., Ph.D., Director, San Raffaele Telethon Institute for Gene Therapy (TIGET) will discuss the larger field of genome editing, utilizing the company's HIV studies as a model.  Sangamo collaborators will also be discussing the use of Sangamo zinc finger nucleases in preclinical and research studies of SCID-X1, cancer and Wiskott-Aldrich Syndrome.

Earlier in October, Geoffrey Nichol, M.B., Ch.B., Sangamo's executive vice president, research and development, was invited to deliver the Foundation Lecture, reviewing recent clinical data from the SB-728-T program at the Autumn Conference of the British HIV Association which was held in London, UK.

Sangamo's SB-728-mR-1401 trial is an open-label, multi-center study designed primarily to evaluate safety and tolerability and the effect of repeat doses of SB-728-T following optimal cyclophosphamide (Cytoxan®) pre-conditioning, on engraftment, viral load and total CD4 counts in peripheral blood.  Electroporation of mRNA is being used to deliver the zinc finger nucleases to  a subject's T-cells to generate the modified autologous T-cell product (SB-728-T). mRNA delivery is more efficient than the previous adenoviral delivery method used and enables treatment of subjects with multiple doses of CCR5-modified cells. Up to nine subjects will be enrolled into two cohorts. Each subject will receive a total of up to 40 billion ZFN modified T-cells. The first cohort will receive this dose divided into infusions of two equal doses of SB-728mR-T 14 days apart after a cyclophosphamide (1 g/m²) preconditioning treatment  two days prior to the first SB-728mR-T infusion, and the second cohort will receive three doses of cells. Dividing the total cell dose and administering sequentially in this manner is thought to maximize overall cell engraftment. Four weeks after the last SB-728-mR infusion, subjects with CD4 cell counts ≥500 cells/mm³ will undergo a 16 week treatment interruption (TI) during which time their anti-retroviral therapy will be discontinued.    

About SB-728-T
Sangamo's therapy, SB-728-T, is generated by ZFN-mediated modification of the gene encoding CCR5 in a patient's own T-cells. ZFN modification disrupts the expression of this key co-receptor for HIV entry and renders cells resistant to HIV infection. The approach is based on the observation that a naturally occurring mutation in the CCR5 gene, CCR5 delta-32, provides protection from HIV infection. Individuals in whom both copies of the CCR5 gene carry the delta-32 mutation are generally not susceptible to the most common strain of HIV.

About Sangamo
Sangamo BioSciences, Inc. is focused on Engineering Genetic Cures™ for monogenic and infectious diseases by deploying its novel DNA-binding protein technology platform in 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 (SB-728-T) and NGF-AAV for Alzheimer's disease (CERE-110). Sangamo's other therapeutic programs are focused on monogenic and rare diseases.  The Company has formed a strategic collaboration with Shire International GmbH to develop therapeutics for hemophilia, Huntington's disease and other monogenic diseases, and with Biogen Idec for hemoglobinopathies, such as sickle cell disease and beta-thalassemia. It has also 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, 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 or control for HIV/AIDS, the ability of a ZFP Therapeutic to control HIV infection, the initiation of clinical studies and enrollment of a Phase 2 clinical trial for HIV/AIDS, projected timing of release of SB-728-T clinical data, clinical data from new HIV/AIDS trials to support pivotal studies, the efficiency of the mRNA delivery system, and the use of Sangamo's technology in potential preclinical and research studies. 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 public filings with the Securities and Exchange Commission, including the risk factors described in its Annual Report on Form 10-K and its most recent Quarterly Report on Form 10-Q. Sangamo assumes no obligation to update the forward-looking information contained in this press release.

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SOURCE Sangamo BioSciences, Inc.

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