26 August 2025

OCU400 for treating retinitis pigmentosa in children and adults

OCU400 is in development for the treatment of retinitis pigmentosa (RP). RP is an inherited eye condition that affects the cells that line the back of the eye in the region known as the retina (the light-sensitive layer at the back of the eye). RP is one of the most complicated genetic conditions, and over 80 causative genes have been identified. 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 RP.

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