The company, based in Malvern, Pennsylvania, reported that the independent Data and Safety Monitoring Board (DSMB) involved in the Phase I/II trial of OCU400, a gene therapy in development for Retinitis Pigmentosa (RP), recommended the study proceed with enrolling more patients. The trial is evaluating the modifier gene therapy OCU400 for RP caused by mutations in the nuclear receptor subfamily 2 group E member 3 (NR2E3) and Rhodopsin (RHO) genes. Ocugen recently dosed the first patient in the study. The DSMB recommended the company continue enrolling the remaining subjects in the current cohort at the target dose level.
RP is a family of rare eye diseases affecting the retina. RP results in the retina breaking down over time, causing vision loss. Symptoms typically begin in childhood, but the majority of patients eventually lose most of their vision. The most common early symptom is loss of night vision.
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