A novel gene therapy for X-linked retinitis pigmentosa (RP)

X-linked Retinitis Pigmentosa (RP) is an inherited retinal disease that starts to appear in childhood, with symptoms like night blindness, progressive loss of peripheral vision and eventual loss of central vision. X-linked RP results from mutations in the RPGR gene, which accounts for 10% of RP cases.

At the moment there is only one available treatment for X-linked RP, the voretigene neparvovec-rzyl (Luxturna, Spark Therapeutics) for RPE65, which represents about 1% of RP. However, a novel new gene therapy is being developed by AGTC-501 (Applied Genetic Technologies Corporation), and recently passed the safety tests of clinical phases 1 and 2.  Read more about the phases of clinical trials here.

Dr Robert A. Sisk, director of Pediatric Vitreoretinal Surgery and Director of Ophthalmic Genetics at Cincinnati Children’s Hospital and the Cincinnati Eye Institute, Ohio, United States said that “The 12-month results of the study of AGTC-501 to treat X-linked RP showed acceptable safety outcomes and vision and microperimetry improvements at higher doses’’.

There were no serious adverse events related to the treatment. The clinical trials of AGTC-501 for the potential treatment of X-linked RP will continue.