Transforming Platform Expertise Into Clinical Data

Targeting unmet medical needs with genomic therapies

Therapeutic Programs Under Development

By functioning at the DNA level our technologies enable us to design therapeutics to address unmet medical needs in monogenic diseases and to potentially achieve long lasting therapeutic outcomes, or even cures, with a single administration.

Therapeutic Programs Under Development
Legend
An icon representing Gene Therapy Gene Therapy
An icon representing Genome Editing Genome Editing
An icon representing Cell Therapy Cell Therapy
An icon representing Gene Regulation Gene Regulation
This table outlines all of the currently active programs in our pipeline.
Lead Indication Approach Program Research Preclinical Phase 1/2 Phase 3
AAV cDNA SB-525
Research Phase complete
Preclinical Phase complete
Phase 1/2 Phase in progress
Phase 3 Phase not started

Hemophilia A is a blood clotting disorder caused by defective clotting Factor VIII gene. Sangamo Therapeutics recently announced that its Investigational New Drug (IND) application for SB-525 had been cleared by the U. S. Food and Drug Administration (FDA). SB-525 is a gene therapy approach to the treatment of hemophilia A that uses a Factor 8 cDNA AAV. The company expects to begin a Phase 1/2 clinical trial in 2017.

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ZFN (insert) SB-FIX
Research Phase complete
Preclinical Phase complete
Phase 1/2 Phase in progress
Phase 3 Phase not started

Hemophilia B is a blood clotting disorder caused by a defective clotting Factor IX gene. We have initiated an open-label, dose-escalating Phase 1/2 clinical trial to assess the safety, tolerability, and preliminary efficacy of SB-FIX. SB-FIX uses our ZFN-mediated in vivo genome editing to place a normal functioning copy of the Factor 9 gene under the control of the strong albumin promoter in the patient’s liver.  This enables the liver to produce and secrete active Factor IX into the bloodstream. SB-FIX is the first in vivo genome editing product to enter the clinic.

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ZFN (insert) SB-318
Research Phase complete
Preclinical Phase complete
Phase 1/2 Phase in progress
Phase 3 Phase not started

MPS I (Scheie, Hurler-Scheie and Hurler syndromes) is a lysosomal storage disorder caused by a deficiency of the enzyme alpha-L iduronidase (IDUA) and results in the toxic buildup of glycosaminoglycans (GAGs). Sangamo Therapeutics has an open IND application for SB-318 and is initiating an open-label, dose-escalating Phase 1/2 clinical trial to assess the safety, tolerability and preliminary efficacy of the treatment. SB-318 uses ZFN-mediated in vivo genome editing to place a normal functioning copy of the IDUA gene under the control of the strong albumin promoter in the patient’s liver.  This enables the liver to produce and secrete active IDUA into the bloodstream to be taken up by other tissues.

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ZFN (insert) SB-913
Research Phase complete
Preclinical Phase complete
Phase 1/2 Phase in progress
Phase 3 Phase not started

MPS II (Hunter syndrome) is a lysosomal storage disorder caused by a deficiency of iduronate-2-sulfatase (IDS) that results in the buildup of glycosaminoglycans (GAGs). Sangamo Therapeutics has an open IND application for SB-913 and is initiating an open-label, dose-escalating Phase 1/2 clinical trial to assess the safety, tolerability and preliminary efficacy of the treatment. SB-913 uses our ZFN-mediated in vivo genome editing to place a normal functioning copy of the IDS gene under the control of the strong albumin promoter in the patient’s liver. This enables the liver to produce and secrete active IDS into the bloodstream to be taken up by other tissues.

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ZFN (knockout) Bioverativ
Research Phase complete
Preclinical Phase in progress
Phase 1/2 Phase not started
Phase 3 Phase not started

Beta thalassemia is a blood disease caused by a mutation in the beta-globin gene that results in greatly impaired production of healthy red blood cells. In collaboration with Bioverativ, we are developing cell therapies for both sickle cell disease and beta thalassemia based on the use of our ZFN genome editing technology to modify a patient’s own (autologous) hematopoietic stem cells (HSCs).

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ZFN (knockout) Bioverativ
Research Phase complete
Preclinical Phase in progress
Phase 1/2 Phase not started
Phase 3 Phase not started

Sickle cell disease is a blood disease caused by a mutation in the beta-globin gene that causes the red blood cells to form an abnormal sickle or crescent shape. The cells are fragile and deliver less oxygen to the body’s tissues resulting in pain and irreversible organ damage. In collaboration with Bioverativ, we are developing cell therapies for both sickle cell disease and beta thalassemia based on the use of our ZFN genome editing technology to modify a patient’s own (autologous) hematopoietic stem cells (HSCs).

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ZFP TF (repress) Shire
Research Phase complete
Preclinical Phase in progress
Phase 1/2 Phase not started
Phase 3 Phase not started

Huntington’s disease is an inherited, progressive neurologic disease for which there is no treatment or cure.  The disease is caused by a mutation in the HTT gene. In collaboration with Shire, Sangamo Therapeutics is developing ZFP Therapeutics that can selectively repress the expression of the mutant disease causing form of HTT while leaving expression levels of the normal gene unchanged. Preclinical studies in animal models of the disease are ongoing, and Shire is responsible for all clinical development activities including filing the IND application.

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Research Phase in progress
Preclinical Phase not started
Phase 1/2 Phase not started
Phase 3 Phase not started

Sangamo Therapeutics is using its zinc finger protein transcription factor (ZFP-TF)-mediated gene regulation technology to develop potential one-time treatments for neurodegenerative diseases associated with tau protein aggregation, including Alzheimer’s disease and other tauopathies. Sangamo intends to seek a partner with CNS disease area expertise for the development and commercialization of its therapeutic gene regulation approach to these indications.

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Various Multiple
Research Phase in progress
Preclinical Phase not started
Phase 1/2 Phase not started
Phase 3 Phase not started

Sangamo Therapeutics is using its ZFN-mediated genome editing technology in T-cells to design improved allogeneic oncology therapies.

Legacy Clinical Research

This table outlines all of the Legacy Clinical Research
Lead Indication Approach Program Research Preclinical Phase 1/2 Phase 3
ZFN (knockout) SB-728-T
Research Phase complete
Preclinical Phase complete
Phase 1/2 Phase in progress
Phase 3 Phase not started

Sangamo Therapeutics' approach uses ZFN-mediated genome editing in T-cells to replicate a naturally occurring human mutation which renders individuals largely resistant to infection with the most common strain of HIV. Sangamo has an open label Phase 2 clinical study to evaluate safety and efficacy of SB-728-T in T-cells.

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ZFN (knockout) SB-728-HSPC
Research Phase complete
Preclinical Phase complete
Phase 1/2 Phase in progress
Phase 3 Phase not started

Sangamo Therapeutics’ approach uses ZFN-mediated genome editing in HSCs to replicate a naturally occurring human mutation which renders individuals largely resistant to infection with the most common strain of HIV. An investigator sponsored open label Phase 1/2 clinical study to evaluate safety and efficacy of SB-728-HSPC in stem cells.

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