Sangamo BioSciences has announced positive preclinical animal efficacy data from its program to develop a ZFP Therapeutic for age-related "wet" macular degeneration (AMD). The study demonstrated the efficacy of zinc finger DNA-binding protein transcription factors (ZFP TF) in an animal model of macular degeneration.
A mouse model of AMD was used to test a ZFP TF designed to "turn on" the gene for PEDF, the eye's key natural regulator of normal blood vessel growth and a neuro-protective agent for the photoreceptors (cells in the eye that respond to light). Mice were treated with the ZFP TF activator of PEDF or a control administered into the vitreous space of the eye and six weeks later a laser was used to induce choroidal neovascularization (CNV), or abnormal blood vessel formation, in the eye. After a further two weeks the size of the laser-induced CNV was measured in both ZFP TF and control-treated eyes and compared. ZFP TF treatment resulted in a statistically significant reduction in CNV compared to control.