X-Linked Retinitis Pigmentosa

X-linked retinitis pigmentosa (XLRP) is inherited condition that causes progressive vision loss in boys and young men.  The condition begins with night blindness in young boys followed by progressive constriction of the field of vision.  Affected men become legally blind at an average of about 45 years of age.

The most common form of XLRP is caused by mutations in the RPGR gene. In August 2017, AGTC announced the filing of an Investigational New Drug application with the U.S. Food and Drug Administration to conduct a Phase 1/2 clinical trial of the company's gene therapy product candidate for the treatment of XLRP caused by mutations in the RPGR gene.

AGTC plans to initiate a clinical study evaluating the safety and efficacy of its proprietary XLRP gene therapy candidate in the United States in the coming months.

For information about AGTC’s XLRP program and/or the upcoming clinical trial, please contact us at advocacy@agtc.com.

Approximately 50 percent of RP cases are isolated and have no previous family history; the cause of these cases cannot be explained. Other cases of RP, where family history has been determined, fall into three main categories: autosomal recessive, autosomal dominant, and X-linked. 

Related Publications

Evaluation of AAV2tYF-GRK1-RPGR Vectors in a Canine Model of RPGR-XLRP

Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa. 

RPGR-associated retinal degeneration in human X-linked RP and a murine model. 

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