Sangamo BioSciences Announces FDA Clearance Of Investigational New Drug Application For SB-525 Gene Therapy Program For Hemophilia A

RICHMOND, CA, USA I January 5, 2017 I Sangamo BioSciences, Inc. (SGMO) today announced that the U.S. Food and Drug Administration (FDA) has cleared the Company's Investigational New Drug application (IND) for its SB-525 gene therapy program for the treatment of  hemophilia A. The IND is now active and enables clinical development to assess the safety, tolerability and potential efficacy of SB-525 in adults with hemophilia A.

"We are very pleased to begin 2017 with the announcement of an open IND for our SB-525 cDNA gene therapy and intend to initiate a clinical trial evaluating SB-525 as soon as possible," said Sandy Macrae, M.B., Ch.B., Ph.D., CEO of Sangamo. "We are committed to developing the best therapeutic options for patients, and based on non-human primate studies, SB-525 has demonstrated the potential to be the best-in-class treatment for Hemophilia A."

SB-525 is one of four lead development programs for which Sangamo is planning to conduct clinical trials in 2017. Sangamo is evaluating SB-FIX, an in vivo genome editing therapy for hemophilia B, in a Phase 1/2 clinical trial, with sites currently screening patients for the study. This year Sangamo will also conduct two Phase 1/2 clinical trials evaluating in vivo genome editing therapies for lysosomal storage disorders MPS I (SB-318) and MPS II (SB-913).

About SB-525
Sangamo's SB-525 gene therapy program uses an adeno-associated virus (AAV2/6) cDNA human Factor 8 construct driven by the Company's proprietary synthetic liver specific promoter, which in preclinical studies appears to be significantly more potent than existing AAV-based cDNA constructs currently under evaluation for the treatment of hemophilia A. The high potency of this novel therapeutic may enable clinically relevant levels of Factor VIII protein to be obtained using lower vector doses, which potentially provides a better therapeutic benefit/risk profile for patients.

About Hemophilia A
Hemophilia, a rare bleeding disorder in which the blood does not clot normally, is caused by mutations in genes that encode factors which help the blood clot and stop bleeding when blood vessels are injured. Hemophilia A is caused by a defect in the gene encoding Factor VIII protein, which is involved in clotting, and individuals with this mutation experience bleeding episodes after injuries and spontaneous bleeding episodes that often lead to destructive joint disease. According to the U.S. Centers for Disease Control and Prevention, hemophilia A occurs in about one in 5,000 live male births. There are about 16,000 people living with hemophilia A in the U.S., with more than half of patients having the severe form of the disease. The standard treatment for individuals with hemophilia is replacement of the defective clotting factor with regular, often frequent infusions of recombinant clotting factors or plasma concentrates. These therapies are expensive and sometimes stimulate the body to produce antibodies against the factors that inhibit the benefits of treatment. The most severe forms of hemophilia A require the need for ongoing, preventive infusions.

About Sangamo
Sangamo BioSciences, Inc. is focused on translating ground-breaking science into genomic therapies that transform patients' lives using the company's industry leading platform technologies in genome editing, gene therapy, gene regulation and cell therapy. The Company's proprietary zinc finger nuclease (ZFN) in vivo genome editing approach is being evaluated in Phase 1/2 clinical trials to treat hemophilia B and lysosomal storage disorders MPS I and MPS II. Sangamo is also conducting a Phase 1/2 clinical trial to evaluate its AAV cDNA human Factor 8 gene therapy approach, SB-525, to treat hemophilia A. Sangamo has a strategic collaboration with Bioverativ, Inc. for hemoglobinopathies, including sickle cell disease and beta-thalassemia, and with Shire plc to develop therapeutics for Huntington's disease. In addition, Sangamo has Phase 1/2 and Phase 2 clinical programs in HIV/AIDS (SB-728). 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

SOURCE: Sangamo BioSciences

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