X-Linked Retinitis Pigmentosa (XLRP)
XLRP is an inherited retinal disease causing significant vision loss in males. Females are usually unaffected carriers, but sometimes experience vision loss from the condition. XLRP is usually diagnosed in childhood or young adulthood, leading to legal blindness by age 45. Early symptoms include night blindness and loss of peripheral vision. Loss of visual acuity occurs at later stages. About 70 percent of cases are caused by mutations in the gene RPGR.
Applied Genetic Technologies Corporation (AGTC) is planning a clinical study at five clinical centers in the U.S. to evaluate the safety and efficacy of an investigational gene therapy in patients with XLRP caused by mutations in the RPGR gene.