Sangamo BioSciences To Highlight 2015 Catalysts And Advancement Of ZFP Therapeutic Programs At The 33rd Annual J.P. Morgan Healthcare Conference

Hunter's Disease and Hurler's Disease Named as Sangamo's First Proprietary Programs in Lysosomal Storage Disorders

Jan 12, 2015, 07:00 ET from Sangamo BioSciences, Inc.

RICHMOND, Calif., Jan. 12, 2015 /PRNewswire/ -- Sangamo BioSciences, Inc. (NASDAQ: SGMO) announced that Edward Lanphier, Sangamo's president and CEO, will present an update on the Company's clinical and preclinical ZFP Therapeutic® programs and an overview of Sangamo's business strategy at 11:00 am PT, today, Monday, January 12th, at the 33rd Annual J.P. Morgan Healthcare Conference in San Francisco. 

During his presentation, Mr. Lanphier will provide an update and outline the expected milestones for Sangamo's ongoing proprietary and partnered ex vivo and in vivo ZFP Therapeutic programs. These include:

  • The initiation in the first half of 2015 of a Phase 1 clinical trial of its zinc finger nuclease (ZFN)-modified hematopoietic stem cell approach for the potential cure of beta-thalassemia.  The Company also expects that an investigational new drug (IND) application for this approach in sickle cell disease will be filed by the end of the year. Both programs are partnered with Biogen Idec.
  • In the first half of 2015, Sangamo also expects to initiate a Phase 1 clinical trial of its ZFP Therapeutic, SB-728, in stem cells, which is designed to enable functional control of HIV.  All subjects have been accrued in the Company's ongoing Phase 2 clinical trial (SB-728-1401) of this approach in T-cells, and initial data are expected from this study by the end of 2015.
  • An update on the progress of Sangamo's proprietary programs in the lysosomal storage disorders (LSDs) that use its In Vivo Protein Replacement Platform (IVPRP) approach. The Company expects to file IND applications for its first two programs, in Hunter's and Hurler's disease, by the end of 2015. The IVPRP makes use of a safe-harbor site, the albumin gene locus, which can be edited with ZFNs to accept and express any therapeutic gene.
  • Continued efforts to expand delivery capabilities and potential therapeutic applications of Sangamo's ZFP technology, including the Company's recently announced initiative to develop in vivo RNA delivery of ZFNs to address in vivo gene knockout targets in the liver.  The potential to dose patients with ZFNs in several treatments provides the opportunity to "dose to effect" or to administer the ZFNs until a sufficient level of permanent modification is obtained to provide a life-long therapeutic outcome. 

"We expect 2015 to be an important year of progress and catalysts for Sangamo's ZFP Therapeutic programs including important milestones for our therapeutic genome-editing platform," stated Mr. Lanphier.  "Given the breadth and clinical validation thus far of our ZFP technology platform, we are well positioned to meet our current goals and advance our ZFP Therapeutic pipeline. We look forward to adding to our accomplishments of 2014 by commencing clinical trials of ZFN-genome edited hematopoietic stem cells for HIV and beta-thalassemia.  We also look forward to filing multiple new IND applications in 2015 to begin human clinical studies of ZFP Therapeutic programs.  These include two proprietary programs which use Sangamo's IVPRP for the treatment of the LSDs, Hunter's disease and Hurler's disease." 

The presentation will be webcast live and may be accessed via a link on Sangamo's website in the Investor Relations section under Events and Presentation.  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 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

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 expected timing for initiating clinical trials, filing of INDs and release of clinical trial data, research and development of novel ZFP TFs and ZFNs and therapeutic applications of Sangamo's ZFP technology platform, the potential of Sangamo's ZFP technology to treat HIV/AIDS and a variety of monogenic diseases including Hunter's disease, Hurler's disease and other LSDs, hemophilia A and B, and Huntington's disease, and the ability to expand the ZFP Therapeutic pipeline. 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 safety, tolerability and efficacy of ZFNs and ZFP TFs, 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.

Logo -


SOURCE Sangamo BioSciences, Inc.