Our current focus is a gene therapy program to treat X-linked Retinoschisis (XLRS) which is a monogenic, inherited orphan disease of the eye in males. This causes progressive loss of central and peripheral vision.
The gene of interest is RS1 which encodes retinoschisin, a protein important for the development and maintenance of the retina.
Our gene-therapy progam is focused on replacing the mutated RS1 gene with a healthy RS1 using a viral vector.
Treatment with Carbonic Anhydrase Inhibitors offers some relief in some patients however successful treatment of XLRS remains an unmet medical need.