CG-311

CG-311 is an investigational gene therapy being developed by CureGene for the treatment of X-linked retinitis pigmentosa (XLRP). XLRP is a severe, early-onset hereditary retinal dystrophy that primarily affects males, leading to progressive photoreceptor degeneration, night blindness, and eventual loss of peripheral and central vision. The condition is most commonly caused by mutations in the *RPGR* (Retinitis Pigmentosa GTPase Regulator) gene, which is essential for the maintenance of photoreceptor cilia. CG-311 is designed to deliver a functional copy of the defective gene to the retinal cells to restore protein expression and preserve visual function. As of early 2024, the program is in the IND-enabling stage of development.