Our scientific efforts are thoughtfully focused on serious conditions for which our technology can make a difference

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Genomic medicines have the potential to shift the treatment paradigm for severe diseases from symptom management to lasting cures.

Sangamo’s proprietary zinc finger (ZF) platform uniquely confers a number of advantages and has broad therapeutic potential due to its versatility, specificity and clinical utility.

Zinc finger technologies are composed of a DNA-binding domain, which binds with precision to any sequence of DNA, and various functional domains (such as nucleases and transcription factors) based on therapeutic need. Due to their compact size, ZF technologies are highly vector compatible. Partnering ZF technologies with delivery vectors allows for the creation of ZF-based genomic medicines.

sangamo science - ZF technology

Sangamo’s zinc finger platform can be used to create genomic medicines for ex vivo for cell therapy and in vivo for genome engineering:

ZF technology - ex vivo / in vivo

An example of ex vivo cell therapy application includes CAR-Tregs produced by Sangamo using ZF engineering to address immune-mediated diseases.

selected cells are engineered to create genomic medicines

An example of in vivo genome engineering application includes the use of zinc fingers coupled to transcription factor domains to create genomic medicines that can regulate gene expression in neurological diseases.

ZF-transcription factors can repress or activate a target gene to controlled therapeutic levels in brain cells

Today, Sangamo is in the clinic with first-generation gene therapy in hemophilia A and Fabry disease, and autologous cell therapy in hemoglobinopathies and renal transplant.

View our pipeline

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Our genomic medicine manufacturing capabilities

Our investment in world-class manufacturing capabilities gives us the advantage of greater control over timelines, quality and supply for our robust pipeline.

We have a state-of-the-art cGMP AAV manufacturing facility and trusted CDMO partner that together will support Sangamo’s diverse pipeline now, and in the future.

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