Sangamo BioSciences Highlights 2016 Catalysts at the 34th Annual J.P. Morgan Healthcare Conference

Sangamo Names Fabry Disease as Fourth Lysosomal Storage Disorder Target

Jan 11, 2016, 07:00 ET from Sangamo BioSciences, Inc.

RICHMOND, Calif., Jan. 11, 2016 /PRNewswire/ -- Sangamo BioSciences, Inc. (NASDAQ: SGMO), the leader in therapeutic genome editing, announced that Edward Lanphier, Sangamo's president and chief executive officer, will present an overview of the Company's therapeutic programs and business strategy at 9:30 am PT, on Tuesday, January 12, 2016, at the 34th Annual J.P. Morgan Healthcare Conference. During his presentation, Mr. Lanphier will provide an update on the Company's clinical stage therapeutic genome editing programs and its robust pipeline of preclinical programs.

"Our accomplishments in 2015 have laid a solid foundation for numerous value-creating catalysts in 2016, and we look forward to building on this success in the year ahead," said Mr. Lanphier. "We are initiating a Phase 1/2 clinic trial for our SB-FIX therapeutic candidate for hemophilia B, which will be the first clinical study of our IVPRP approach, and we have achieved our objective of filing an Investigational New Drug (IND) application for our MPS I program in 2015. We are also moving quickly to advance additional ZFP Therapeutic programs into the clinic with the target of filing six more IND applications over the next twelve months. We have begun 2016 with more than $200 million in cash and investments, which provides us with the financial resources to aggressively pursue our goals of moving multiple programs into the clinic to make a fundamental difference in the lives of patients and maximize value for our shareholders."

Sangamo's milestones and objectives for 2016 include:

  • Initiation of the first in vivo genome editing clinical trial. Sangamo expects to begin enrolling subjects into a Phase 1/2 clinical trial for the first application of its In Vivo Protein Replacement Platform™ (IVPRP™) approach to assess the safety, tolerability, and efficacy of SB-FIX as a potentially curative therapy for hemophilia B.
  • Submission of IND applications for the Company's additional IVPRP-based proprietary programs in hemophilia A and lysosomal storage disorders (LSDs). Sangamo expects to file IND applications for MPS II in the first half of 2016 and for hemophilia A, Gaucher disease, and Fabry disease in the second half of 2016.
  • Submission of IND applications from Sangamo's collaborative programs with Biogen to develop one-time, potentially lifelong treatments for beta-thalassemia and sickle cell disease. Sangamo expects to file the first IND application for its Bcl11a Enhancer knockout approach for beta-thalassemia in the first half of 2016. The companies expect to file an IND application for the same approach for the treatment of sickle cell disease in the second half of 2016.
  • Presentation of final data from Sangamo's ongoing Phase 2 clinical trials in HIV. Sangamo expects to present new and updated clinical data from its SB-728-1101 Cohort 3* study in the second half of 2016.
  • Ending 2016 with at least $150 million in cash and investments.

Mr. Lanphier's presentation will be webcast live and may be accessed via a link on the Sangamo BioSciences website in the Investor Relations section under Events and Presentations. The presentation will be archived on the Sangamo website for two weeks after the event.

About Sangamo Sangamo BioSciences, Inc. is focused on Engineering Genetic CuresTM for monogenic and infectious diseases by deploying its novel DNA-binding protein technology platform in therapeutic genome editing and gene regulation. The Company's proprietary In Vivo Protein Replacement Platform™ (IVPRP™) approach is focused on monogenic diseases, including hemophilia and lysosomal storage disorders. In addition, Sangamo has a Phase 2 clinical program to evaluate the safety and efficacy of novel ZFP Therapeutics® for the treatment of HIV/AIDS (SB-728). The Company has also formed a strategic collaboration with Biogen Inc. for hemoglobinopathies, such as sickle cell disease and beta-thalassemia, and with Shire International GmbH to develop therapeutics for Huntington's disease. It 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 achievement of strategic milestones in 2016, the projected cash balance at the end of 2016, the potential of ZFNs to treat a broad range of human monogenic diseases, including beta-thalassemia, sickle cell disease, hemophilia A and B, and lysosomal storage disorders (LSDs), research and development of novel ZFNs, therapeutic applications of Sangamo's ZFP technology platform in hemoglobinopathies, and using its IVPRP in indications such as hemophilia and LSDs, the anticipated timing and the number of IND filings and initiation and completion of clinical trials for IVPRP and collaborative programs with Biogen; and the anticipated timing for the announcement of Phase 2 clinical trial data for the HIV program. 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, and lack of control in therapeutic programs developed under collaborative agreements. 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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