Sangamo is a clinical stage biopharmaceutical company focused on the research, development and commercialization of engineered DNA-binding proteins for the development of novel therapeutic strategies for unmet medical needs.
We are worldwide leaders in the development of a proprietary technology platform that enables specific regulation of gene expression and gene modification. The basis of this platform is a naturally occurring class of transcription factors, zinc finger DNA-binding proteins (ZFPs) which we can engineer to drive desired therapeutic outcomes. Engineered ZFPs can be linked to functional domains that normally activate or repress gene expression to create ZFP transcription factors (ZFP TFs) capable of turning genes on or off. We can also link ZFPs to nuclease domains to create zinc finger nucleases (ZFNs) which enable precise gene-editing in cells. Engineered ZFNs can modify a cell's DNA at a precise location, thereby facilitating correction or disruption of a specific gene or the targeted addition of a new DNA sequence.
Our primary mission is to develop ZFP Therapeutics®. We have ongoing clinical programs to evaluate ZFP TFs and ZFNs as novel approaches to unmet medical needs where we believe we have a differential technical advantage to impact the outcome of disease by functioning at the DNA level.