OCU100

OCU100 is a recombinant truncated N‑terminal fragment of human lens epithelium‑derived growth factor comprising amino acids 1‑326 (LEDGF1‑326), developed as a biologic therapy for inherited retinal degenerations, particularly retinitis pigmentosa. It is designed to act as a cytoprotective growth factor in retinal cells, reducing misfolded protein aggregation and associated cellular stresses in photoreceptors and retinal pigment epithelium, thereby aiming to preserve retinal structure and function in a gene‑agnostic manner.[1][3][10] The program originated from academic research at the University of Colorado Anschutz Medical Campus and has been advanced by Ocugen, which has received FDA Orphan Drug Designation for OCU100 in retinitis pigmentosa and has described plans for early‑stage clinical development.[3][5][8][10][11][12]

Other names

Recombinant lens epithelium-derived growth factor 1-326Lens epithelium-derived growth factor (1-326)LEDGF(1-326)Recombinant truncated N-terminal fragment of human lens epithelium-derived growth factor