We have several ZFP Therapeutic® programs under development. By functioning at the DNA level our technology enables us to achieve permanent biologic outcomes in the treatment of monogenic and infectious diseases with unmet medical needs.
We are using engineered zinc finger DNA-binding protein transcription factors (ZFP TFs) to up-regulate (activate) or down-regulate (repress) gene expression to achieve a desired therapeutic effect. We are also using zinc finger nucleases (ZFNs) to insert or knockout 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 hemoglobinopathies, beta-thalassemia and sickle cell disease, in collaboration with Biogen, and hemophilia A and B and Huntington's disease in partnership with Shire: