OCU400

OCU400 is a gene therapy designed as a gene-agnostic modifier therapy for inherited retinal diseases, particularly retinitis pigmentosa (RP) and Leber congenital amaurosis. It utilizes an adeno-associated viral vector to deliver the human nuclear hormone receptor subfamily 2 group E member 3 (NR2E3) gene to retinal cells. NR2E3 regulates key physiological functions in the retina, including photoreceptor development and maintenance, metabolism, phototransduction, inflammation control, and cell survival. By restoring or modulating these pathways regardless of the underlying genetic mutation causing RP or related disorders, OCU400 aims to reset dysfunctional gene networks and re-establish cellular homeostasis in retinal cells. This approach offers potential benefit across multiple genetic forms of RP where no approved therapies currently exist[1][4][5][6][7].