Inherited retinal diseases are caused by a mutation in one or more genes, which prevents the synthesis of functional proteins necessary for healthy vision; for example, loss of function mutations in the CHM gene leads to the progressive degeneration of the retinal pigment epithelium, photoreceptors, and choriocapillaris, a condition called choroideremia, while mutations in the RPE65 gene can cause Retinitis Pigmentosa 20, which leads to the degeneration of rods and cones, then, subsequently, the retinal pigment epithelium. Such diseases typically onset in childhood, however there is a therapeutic window when it is possible to administer a therapy and preserve vision.

Retinal cells do not replicate, making it a good target for life-altering gene therapy by preventing disease progression in affected patients. There have already been successful instances of gene therapies being developed. Artema specializes in the development of such therapies, with the goal of preserving the vision and quality of life of our patients.