opct-001

OpCT-001 is an investigational allogeneic cell therapy composed of human induced pluripotent stem cell (iPSC)-derived photoreceptor cells. It is designed to treat primary photoreceptor diseases, a subgroup of inherited retinal disorders that includes retinitis pigmentosa and cone-rod dystrophy. These conditions are characterized by degeneration and loss of photoreceptor cells in the retina, leading to irreversible vision loss in both children and adults. OpCT-001 works by replacing degenerated retinal photoreceptors with functional iPSC-derived cones and rods, aiming to restore visual function. The therapy is administered via subretinal injection. OpCT-001 is the first iPSC-derived cell therapy to be clinically tested for these indications and has received FDA Fast Track designation due to its potential to address a significant unmet medical need[1][2][3][5].