Sangamo BioSciences Presents New Data From In Vivo Protein Replacement Platform For Development Of ZFP Therapeutics® For Monogenic Diseases

Production of Therapeutically Relevant Levels of Clotting Factor VIII Further Demonstrates Breadth and Leverage of this Novel Approach to Monogenic Diseases

Presentations at ASGCT Annual Meeting Highlight Broad Applicability of ZFP Technology

May 20, 2013, 07:00 ET from Sangamo BioSciences, Inc.

RICHMOND, Calif., May 20, 2013 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced the presentation of new data demonstrating the successful application of Sangamo's proprietary In Vivo Protein Replacement Platform (IVPRP) to produce therapeutically relevant levels of Factor VIII in a mouse model.  Sangamo has partnered with Shire AG (LSE: SHP, NASDAQ: SHPG) to develop ZFP Therapeutics for both hemophilia A and B using this approach. The data were presented at the 16th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) which was held in Salt Lake City from May 15-18, 2013.


"Our IVPRP provides a potentially curative therapeutic solution for hemophilia A – a monogenic disease caused by the absence of the coagulation protein, Factor VIII," said Philip Gregory. D. Phil., Sangamo's vice president of research and CSO. "These data provide further proof-of-concept for the broad application of our IVPRP strategy for monogenic diseases currently treated using protein replacement.  Our data demonstrate that a single systemic treatment results in the stable production of therapeutically relevant levels of functional Factor VIII."

Hemophilia A, the most common form of the bleeding disorder, is caused by a genetic defect in the gene encoding Factor VIII, a blood clotting factor.  Sangamo's IVPRP approach enables the precise insertion of a replacement Factor VIII gene into the genome at what is known as a "safe harbor site." Specifically, Sangamo's IVPRP uses the albumin gene as the site of insertion ensuring that Factor VIII will be highly expressed at stable levels exclusively in the liver.

Based on Sangamo's zinc finger DNA-binding protein (ZFP) genome-editing technology, the IVPRP enables the permanent production of therapeutic proteins from the liver with a single systemic treatment, potentially providing curative treatments for a range of monogenic diseases including hemophilia and lysosomal storage disorders (LSD) such as Gaucher and Fabry disease. Such diseases are currently treated by regular infusions of protein or enzyme replacement therapy (ERT) throughout the patient's life. 

Data were presented on the production of Factors VIII and IX for the correction of hemophilia A and B, as well as four enzymes absent in a panel of LSDs by Sangamo's collaborators from the laboratory of Katherine A. High, M.D., director of the Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia, and Investigator, Howard Hughes Medical Institute.  The abstract for this presentation, entitled "In Vivo ZFN Mediated Targeting of Albumin as a Platform for Expression of Multiple Therapeutic Genes," was chosen as one of the six Excellence in Research Awards presented at the meeting.

"The ASGCT meeting provides a great forum to highlight our latest data demonstrating the potential of our ZFP platform to provide novel therapeutic solutions to a broad range of diseases," said Edward Lanphier, Sangamo's president and CEO. "Our presentations included data from clinical and preclinical programs to develop ZFP Therapeutics for indications including HIV/AIDS, hemophilia A and B, lysosomal storage disorders and other monogenic diseases, and cancer. 

In one of the first sessions of the ASGCT meeting, we presented data from our SB-728-T clinical trials in HIV-infected subjects demonstrating unprecedented increases in total CD4 T-cells and reduction in HIV reservoir as measured by proviral DNA, and encouraging preliminary data from our ongoing studies.  Data demonstrating the successful production of Factor VIII demonstrates the unique qualities of our IVPRP approach to deliver potentially curative solutions for many monogenic diseases. We look forward to presenting further data from these programs and others in our ZFP Therapeutic pipeline at scientific and medical meetings throughout the year."

A total of thirteen data presentations were made by Sangamo scientists and their collaborators at the 16th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) which was held in Salt Lake City from May 15-18, 2013. 

Presentations included data from the following programs and collaborations in the following areas:


  • Clinical Studies of the Infusion of ZFN CCR5 Modified Autologous CD4 T cells (SB-728T) in HIV Subjects, presented by Dale Ando, M.D. who also chaired the session entitled, Challenges and Success of Gene Therapy Product Approval.
  • Long Term CD4 Reconstitution in HIV Subjects Receiving ZFN CCR5 Modified CD4 T-Cells (SB-728-T) May Be Attributed to the Sustained Durability of the Central Memory T-Cell Subset. (Abstract #58)
  • T-Cells Edited To Express the C34 Peptide from gp41 Heptad Repeat-2 Fused to CCR5 or CXCR4 Exhibit Robust Protection from Diverse HIV-1 Isolates. (Abstract #46)
  • Next Generation TALENs Mediate Efficient Disruption of the CCR5 Gene in Human HSCs. (Abstract # 184)

Monogenic Diseases

  • In Vivo ZFN Mediated Targeting of Albumin as a Platform for Expression of Multiple Therapeutic Genes. (Abstract #488)
  • Allele-Preferred Targeted Correction of CFTR Gene in Cystic Fibrosis Induced Pluripotent Stem Cells. (Abstract # 107)
  • Rescue of T-cell deficiency in Prkdc SCID mice by transplantation of gene-repaired haematopoietic stem cells. (Abstract # 678)


  • TCR Gene Editing To Treat Acute Leukemia and Multiple Myeloma. (Abstract #504)

Technology Development

  • Electroporation of ZFN mRNA Enables Efficient CCR5 Gene Disruption in Mobilized Blood Hematopoietic Stem Cells at Clinical Scale. (Abstract #183)
  • Efficient Site-Specific Integration and In Situ Gene Correction of Human Long-Term Repopulating Hematopoietic Stem Cells by Zinc Finger Nucleases. (Abstract #486)
  • Optimizing Plasmid DNA Templates for Homologous Recombination-Mediated Gene Modification Following Zinc Finger Nuclease Treatment of Hematopoietic Stem Cells. (Abstract #487)
  • Enhancing the Efficiency of Zinc Finger Nuclease Delivery by Integrase-Defective Lentiviral Vectors Using Valproic Acid. (Abstract #492)
  • In Vivo Cleavage of Transgene Donors Promotes Nuclease-Mediated Targeted Integration. (Abstract#568)

All abstracts for the meeting are available online at 2013 ASGCT Meeting Abstracts.

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 and  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, Huntington's disease 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, the research and development of novel ZFP TFs and ZFNs as ZFP Therapeutics and therapeutic applications and the scope of such applications of Sangamo's ZFP technology platform to specific human diseases and unmet medical needs, including a potential functional cure of HIV/AIDS, the expansion of clinical studies of SB-728-T in HIV-infected individuals, expected timing for the presentation of clinical trial data, the development of ZFP Therapeutics for hemophilia and other monogenic diseases 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 the 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.