AGN-001

AGN-001 is an off-the-shelf, induced pluripotent stem cell (iPSC) organoid-derived cell therapy being developed by Agnos Therapeutics for the treatment of inherited blindness and inherited retinal degeneration. The therapy is designed to be mutation-agnostic, intended to work across many of the 270+ gene mutations linked to retinal disease. AGN-001 is administered via local delivery into the subretinal space, where the transplanted cells reside adjacent to host cells and deliver healthy proteins to durably rescue sight. Preclinical studies have demonstrated measurable visual improvement within one week of treatment in animal models. The therapy is cryopreserved and ready to use, requiring no patient-specific preparation.