Target Dry AMD
Only one therapy exists for the dry form of AMD, which – however — concerns about 90% of patients suffering from AMD in total. This drug, Syfovre®, is currently available only in the United States and shows a similar administration route and frequency as drugs for wet AMD, and thus, shows comparable drawbacks. Dry AMD is caused by inflammatory reactions and oxidative stress that results in the accumulation of waste products secreted by the Retinal Pigment epithelial (RPE) cells. These so called drusen further damage the RPE cells until they undergo cell death. The weakened and lost Retinal Pigment epithelial cell layer has two main consequences: 1) Important neuroprotective factors are insufficiently produced and 2) photoreceptors lose contact with Retinal Pigment epithelial cells. Both lead to damage up to the death of photoreceptors and thus, in the advanced stage, large deteriorated, cell-less (atrophic) areas of the retina.
Based on the TargetAMD 1.0 approach, we aim to transplant subretinally autologous Iris Pigment Epithelial cells genetically modified with a combination of two therapeutic genes, one coding for a cell protective factor (Pigment Epithelium-Derived Factor) and one against inflammation and oxidative stress (Granulocyte Macrophage Colony-Stimulating Factor). The continuous delivery of these proteins to the retina shall prevent further loss and degeneration of the retinal Pigment epithelial and the retinal cells.
Definition: Oxidative stress is a term for biochemical reactions induced by the processing of oxygen. These processes occur permanently in our body and particularly in the retina. In a healthy environment, harmful byproducts of the reactions are eliminated. In age and disease, however, this so-called antioxidant defense is weakened, harmful byproducts accumulate and damage cells.