Sangamo BioSciences Announces FDA Acceptance of IND to Initiate Clinical Trial of its Novel ZFP Therapeutic® for Beta-Thalassemia

RICHMOND, Calif., Feb. 4, 2015 /PRNewswire/ -- Sangamo BioSciences, Inc. (NASDAQ: SGMO) announced today that an Investigational New Drug (IND) application for the company's SB-BCLmR-HSPC genome editing approach, which is designed to provide a  one-time lasting therapy for beta-thalassemia, has been accepted by the U.S. Food and Drug Administration (FDA) and is now active. This enables Sangamo to initiate a Phase 1/2 clinical trial of the ZFP Therapeutic in transfusion-dependent patients with beta-thalassemia major.  The trial, which is expected to begin in 2015, is designed to assess the safety and tolerability, and measures of efficacy of this novel approach. Sangamo is developing the therapy in collaboration with Biogen Idec.

"We believe that a single treatment with SB-BCLmR-HSPC has the potential to provide a lasting therapeutic solution  for transfusion-dependent beta-thalassemia with significant safety advantages over existing transplant therapies that involve hematopoietic stem progenitor cells (HSPCs) from a matched related donor," said Geoff Nichol, M.B., Ch.B., Sangamo's executive vice president of research and development. "We are using our genome editing technology to target a key genetic switch in a patient's own HSPCs to enable continued production of fetal hemoglobin in the red blood cells of adults.  We know that elevated production of fetal globin can ameliorate disease symptoms of hemoglobinopathies such as beta-thalassemia.  We are also developing this strategy for sickle cell disease."

In May 2013, Sangamo was awarded a $6.4 million Strategic Partnership Award from CIRM, California's stem cell agency, to develop a ZFP Therapeutic for beta-thalassemia. The four-year grant provided matching funds for preclinical work to support the IND application and for a Phase 1/2 clinical trial, which will be carried out at multiple centers, including UCSF Benioff Children's Hospital Oakland.

"Our research and development team has worked hard to rapidly advance SB-BCLmR-HSPC through preclinical development and the IND application process," said Edward Lanphier, Sangamo's president and CEO. "We are very pleased to be in a position to move this therapy into the clinic with the aim of providing transfusion-dependent beta-thalassemia patients with a one-time treatment for this devastating disease."

Sangamo's ZFP Therapeutic^® for Hemoglobinopathies
Sangamo's proprietary ZFN genome editing technology enables multiple approaches to the correction of beta-thalassemia and sickle cell disease (SCD). Both diseases manifest after birth, when patients switch from producing functional fetal gamma-globin to a mutant form of adult beta-globin, which results in their condition. Naturally occurring increased levels of fetal hemoglobin have been shown to reduce the severity of both beta-thalassemia and SCD disorders in adulthood. Sangamo's genome editing can be used to precisely disrupt key transcriptional regulators in HSPCs to reverse the switch from expression of the mutant adult beta-globin back to the production of functional fetal gamma-globin.

A bone marrow transplant (BMT), of HSPCs from a "matched" related donor (allogeneic BMT) is curative for both diseases. However, this therapy is limited due to the scarcity of matched donors and the significant risk of Graft versus Host Disease (GvHD) after transplantation of the foreign cells. By performing genome editing in HSPCs that are isolated from and subsequently returned to the same patient, an autologous HSPC transplant, Sangamo's approach eliminates both the need for a matched donor and the risk of acute and chronic GvHD. The ultimate goal of this approach is to develop a one-time treatment for beta-thalassemia.

About Hemoglobinopathies
Mutations in the genes encoding beta-globin, a subunit of the oxygen-carrying protein of red blood cells, lead to the hemoglobinopathies beta-thalassemia and SCD. There are several forms of beta-thalassemia caused by mutations in the beta-globin gene; broadly the disorder results in defective production of red blood cells leading to life-threatening anemia, enlarged spleen, liver and heart, and bone abnormalities. Severe beta-thalassemia major requires regular, often monthly, blood transfusions and subsequent iron-chelation therapy to treat iron overload. The CDC estimates that 2,000 people have beta-thalassemia in the United States, and an unknown number carry the genetic trait and can pass it on to their children.  Thalassemia is most common among people of Mediterranean descent and is also found among people from the Arabian Peninsula, Iran, Africa, Southeast Asia and Southern China. 

About Sangamo
Sangamo BioSciences, Inc. is focused on Engineering Genetic Cures^TM 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, the potential of Sangamo's ZFP technology to treat beta-thalassemia and sickle cell disease, the safety of the approach of using ZFN-mediated genome editing, and Sangamo's collaboration with Biogen for the treatment of hemoglobinopathies, beta-thalassemia and sickle cell disease. 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.

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SOURCE Sangamo BioSciences, Inc.

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