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 our engineered zinc finger nuclease (ZFN) genome editing platform to insert or knockout genes in the genome, and our adeno-associated virus (AAV)-based gene therapy platform to deliver therapeutic genes into cells. Both platforms can be used to address diseases caused by a defect in a single gene, so called “monogenic diseases”. We also use zinc finger protein transcription factors (ZFP TFs) to up-regulate (activate) or down-regulate (repress) gene expression to achieve a desired therapeutic effect.
We are currently developing the following ZFP Therapeutics, including programs in hemoglobinopathies, beta-thalassemia and sickle cell disease, in collaboration with Biogen, and Huntington's disease in partnership with Shire: