Unlocking the potential of genetic cures begins with using the right key
Sangamo has developed a range of capabilities that enable us to address serious and life-threatening genetic diseases with the appropriate therapeutic approach. These include best-in-class capabilities in gene therapy and genome editing, which are being deployed to produce therapeutic proteins from the liver and to generate new types of cell-based therapies. We can also regulate genes for therapeutic benefit.
Sangamo is the world leader in the development of engineered zinc finger proteins (ZFPs) which can be designed for highly specific recognition of any chosen DNA sequence. We use this powerful targeting capability in zinc finger nucleases (ZFNs) to edit the human genome, specifically knocking out a gene or adding a new DNA sequence in a precise location. Using our ZFP transcription factors (ZFP TFs) we can also control the expression of any gene to produce more of a therapeutically relevant protein or down regulate the gene to produce less.
We currently use both adeno associated virus (AAV) and mRNA to deliver our therapeutics and continue to develop and refine these platforms as well as our manufacturing processes for therapeutic cell production. Using our molecular biology and AAV expertise we are also developing gene therapy approaches for key indications.
DNA is DNA – it doesn’t matter what type of cell or species one wants to edit or regulate. ZFPs have applications beyond human therapeutics. This technology can be used in agriculture as well as research applications such as the production of cell lines for manufacturing or drug testing and the development of transgenic animals including disease models.
With our collaborators, we have published foundational research in the fields of genome editing, gene therapy, cell therapy and gene regulation.