Background

The retinal pigment epithelium (RPE) performs many critical functions that support photoreceptor health and integrity, including phagocytosis of outer segments, secretion of a variety of growth factors and transport of nutrients and metabolic end-products from the subretinal space to the blood.

Dysfunction and loss of RPE are major pathologies observed in various degenerative retinal diseases leading to blindness, including Stargardt disease, Best disease and subtypes of Retinitis Pigmentosa. Furthermore, the most common RPE disease is Age-related Macular Degeneration (AMD), in which the RPE ceases to function normally and phagocytose efficiently.

Traditional gene therapies currently being tested are limited to monogenic diseases (i.e. MerTK-associated retinitis pigmentosa) and are facing challenges to capture mutational variability across larger genes (i.e. ABCA4, the