The company is a leader 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 are engineered 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. They 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. Its lead mission is to develop ZFP Therapeutics as novel approaches to unmet medical needs.