Treating Age-Related Macular Degeneration (AMD) With A Novel Stem Cell Therapy

The AGI for regenerative medicine, led by the NEI, seeks new therapies for challenging eye diseases like AMD, the leading cause of vision loss in the U.S. With no approved treatments to slow, stop, or reverse AMD—particularly the more common dry form, affecting 80-90% of cases—researchers are exploring regenerative approaches. In advanced stages, AMD destroys retinal pigment epithelium (RPE) cells, essential for photoreceptor function. A promising strategy involves differentiating induced pluripotent stem cells (iPSCs) into RPE cells and implanting them to restore vision. NEI has developed a novel delivery system using a biodegradable PLGA scaffold to support RPE integration into the retina.

In collaboration with Ocular Services On Demand (OSOD), Labcorp contributed to NEI’s IND submission by conducting preclinical studies in rat models, assessing tolerability, biodistribution, toxicity, and tumorigenicity through two pilot and four GLP studies.