How does QR-1123 work?
QR-1123 aims to prevent vision loss or restore vision in people with retinitis pigmentosa due to the P23H mutation in the RHO gene. This mutation causes a mistake in the RHO RNA and the production of mutated rhodopsin protein. Over time, the mutated protein blocks the function of the normal rhodopsin protein and eventually becomes toxic to the retina.
QR-1123 works by binding to the mutated RHO RNA so that it is removed by the cell through a mechanism called RNase H mediated cleavage. Once the mutated version of the RNA is removed, the normal version of the protein can perform its function in the retina again.