Botaretigene sparoparvovec for treating X-linked retinitis pigmentosa


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Botaretigene sparoparvovec is in development for the treatment of X-linked retinitis pigmentosa. Retinitis Pigmentosa (RP) is an inherited eye condition that affects the cells that line the back of the eye in the region known as the retina. RP is one of the most complicated genetic conditions, and over 80 causative genes have been identified.

Indications: Retinitis pigmentosa
Therapeutic Areas: Ophthalmology
Year: 2023

Botaretigene sparoparvovec is in development for the treatment of X-linked retinitis pigmentosa. Retinitis Pigmentosa (RP) is an inherited eye condition that affects the cells that line the back of the eye in the region known as the retina. RP is one of the most complicated genetic conditions, and over 80 causative genes have been identified. X-linked RP is caused by a defect in a gene called RPGR which is located on the X-chromosome. Symptoms initially include difficulty seeing in dim light, including transitioning from light to dark and vice versa. Peripheral vision will also decline, resulting in a narrowing of the visual field and, in some cases, resulting in complete blindness. There are currently no approved treatments that can effectively slow or stop the progression of X-linked RP.