We have several ZFP Therapeutic® programs under development. By functioning at the DNA level our technology enables us to achieve unique biologic outcomes in the treatment of unmet medical needs.
We are using engineered zinc finger DNA-binding protein transcription factors (ZFP TFs) to "up-regulate" or "down-regulate" gene expression to achieve a desired therapeutic effect. We are also using zinc finger nucleases (ZFNs) to correct or disrupt genes to address diseases caused by a defect in a single gene, so called “monogenic diseases”.
We are currently developing the following ZFP Therapeutics including programs in hemophilia and Huntington's disease with Shire AG.
As part of its acquisition of Ceregene Inc., Sangamo acquired a fully-enrolled, double-blind, placebo-controlled Phase 2 trial to evaluate NGF-AAV (CERE-110) in Alzheimer's disease. CERE-110 is a therapeutic that uses adeno-associated virus (AAV) to deliver the gene for nerve growth factor (NGF) into the brain of subjects with mild to moderate Alzheimers disease with the aim of helping nerve cells survive and function better. Data from the trial will be available in 2015.