Our Technology Focus
Sangamo is focused on the research and commercial development of engineered DNA-binding proteins for the regulation of gene expression and for gene modification. The basis of our proprietary technology is the engineering of a naturally occurring class of transcription factors, zinc finger DNA-binding proteins (ZFPs). When linked to functional domains that normally activate or repress gene expression, we create ZFP transcription factors (ZFP TFs) capable of turning genes on or off. ZFPs can also be linked to nuclease domains to create zinc finger nucleases (ZFNs). Engineered ZFNs can modify genomic DNA at a preselected location facilitating correction or disruption of a specific gene or the targeted addition of new DNA sequence.

A New Class of Therapeutics
Our primary mission is to develop ZFP TF and ZFNs as ZFP Therapeutics™; ZFP TFs designed to directly regulate disease-related genes and ZFNs to disrupt or correct therapeutically relevant genes.
The human genome has been sequenced and genes associated with many diseases are being identified. Using our technology we can regulate the cell’s own genes —turning on the expression of therapeutically-beneficial genes and turning off the expression of disease-related genes. Our ZFP TFs regulate the cellular copy of the gene and mimic the cell’s natural method of gene regulation - turning on or off all of the natural forms of the gene product.
We are also developing our ZFN technology for applications in human therapeutics. We are using ZFNs to correct mutations in genes that cause disease, so-called monogenic diseases such as X-linked severe immunodeficiency (X-linked SCID) and Wiskott-Aldrich Syndrome or blood diseases such as hemophilia. In addition, we are developing ZFNs to disrupt genes that facilitate disease. In our program for HIV/AIDS we are using ZFNs to disrupt CCR5, a co-receptor for HIV. Our goal is to generate a protected population of immune cells that cannot be infected by the virus and are available to fight opportunistic infections and the virus itself.

A Powerful Technology with Broad Applications
Our ZFP TF gene regulation technology can be used to regulate any gene in any cell type. Moreover, as we regulate genes as they naturally occur within cells and do not use copies of those genes, or cDNAs, we are not encumbered by patent restrictions that may cover those cDNAs. Our ZFN gene modification technology can be used to to correct, disrupt or add a sequence of DNA at a specified location in a genome. We are also using this technology to enhance production of protein pharmaceuticals and in applications in laboratory research reagents with Sigma-Aldrich Corporation and plant agriculture with Dow AgroSciences, Inc.

Unique Advantages of Our Technology
Our ZFP technology has several differential technical and business advantages:

ZFP TFs

• Naturally have an important role in gene regulation in all higher organisms. Our engineered ZFP TFs mimic the natural mode of gene regulation used by the cell and enable the production of all of the natural forms of a protein encoded by a single gene.
• Can be used to regulate any gene in any cell type.
• Can be used to turn genes on or off.
• Regulate a cell’s own genes not a copy of that gene and so are not encumbered by cDNA patent restrictions.
• Can be regulated themselves giving more control over their effects.
• Have proven effectiveness from cell models through to the whole organisms.  

ZFNs

• Can be used to corrrect or disrupt genes or add a DNA sequence into a specific location in the genome of any organism.
• Permanent gene correction, disruption or addition requires only transient cellular expression of ZFNs.