Sangamo is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for various monogenic and infectious diseases with unmet medical needs. Their therapeutic products are based on our engineered zinc finger DNA-binding protein (ZFP) genome editing and adeno-associated virus (AAV) gene therapy platforms.
Sangamo is a worldwide leader in the fields of gene therapy and genome editing. Their AAV-based gene therapy platform uses our proprietary vector constructs to enable the delivery and potent expression of any therapeutic gene. Their proprietary ZFP technology platform enables highly specific genome modification and gene regulation. ZFPs are a naturally occurring class of transcription factors, which they can engineer to bind to any DNA sequence with singular specificity and drive desired therapeutic outcomes.
Their 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. Sangamo can also link ZFPs to endonuclease domains to create zinc finger nucleases (ZFNs) which enable precise genome editing in cells. Sangamo’s engineered ZFNs can modify a cell’s DNA at a precise location, thereby facilitating the correction or disruption of a specific gene or the targeted addition of a new DNA sequence, without the unwanted consequences of off-target DNA binding activity.