AMD

AMA003 is a gene therapy product for the treatment of patients with age related macular degeneration (AMD). AMD is the main cause of irreversible impaired vision and blindness in elderly living in industrialized countries. The wet form of the disease is characterized by choroidal neovascularization (CNV) resulting from the growth of abnormal leaky blood vessels from the choroid into the retina. CNV is mediated by increased levels of vascular endothelial growth factor (VEGF) in the retina of AMD patients. RNA interference (RNAi) is a powerful tool to regulate the expression of VEGF and shows great promise for therapeutic applications. Using AMA003 we intend to inhibit the VEGF expression by short hairpin-mediated RNA interference for the treatment of AMD patients.