A new cell therapy for age-related macular degeneration (AMD) is showing early promise in preclinical studies using retinal pigment epithelium (RPE) cells derived from donor eye tissue provided by The Eye-Bank’s newly renamed Patricia Dahl Ocular Tissue Research Program. Preliminary findings were reported in the September 2025 Cell Stem Cell article, “Safety and tolerability of RPESC-RPE transplantation in patients with dry age-related macular degeneration: Low-dose clinical outcomes,” by Rao et al., which demonstrated the safety and tolerability of RPESC-RPE transplantation in a small group of patients with dry AMD.
A related publication in April of this year describes the roadmap of preclinical findings that led to approval of an Investigational New Drug (IND) application to the FDA. This related work was reported by Arduini et al. in Cell Reports Medicine in the article titled “IND-enabling safety and efficacy of RPESC-RPE-4W, an adult RPE progenitor cell therapy for age-related macular degeneration.”
In these articles, co-founders of the Neural Stem Cell Institute Jeffrey H. Stern, M.D., Ph.D., and Sally Temple, Ph.D., along with Eye-Bank Medical Advisory Board Member Timothy Blenkinsop, Ph.D. and their co-authors, described the safety of cultured RPE cells from human donor eyes with an indication of visual acuity improvement in the small number of subjects who received RPE transplantation.
If untreated or unmonitored, AMD can lead to severe vision impairment and vision loss. Retinal pigment epithelium (RPE) cells, the cells damaged by AMD, support photoreceptor cells, which are essential to receive light signals the brain uses for vision. The aim of this work is to not only slow down the damage caused by AMD but to replace damaged or missing RPE cells with new, healthy cells, potentially restoring or improving vision.
“It has been a privilege to support Dr. Stern and Dr. Temple’s research and to work alongside their team,” said Eye-Bank Research & Relationship Development Manager YoneJung Yoon, Ph.D. “These preclinical and clinical studies represent an exciting step forward in the fight against blindness. It is rewarding to know that precious donor tissues gifted by eye donors contributed these valuable cells that may help advance regenerative therapies for vision restoration.”
The Eye-Bank has been collaborating with the Neural Stem Cell Institute since 2021 and with Dr. Stern since 1994.
The Patricia Dahl Ocular Tissue Research Program provides quality donor eye tissue to support advancements in ocular research. The program offers customized “control” and diseased ocular research tissues and human primary ocular cells such as RPE cells to assist with ocular, neurodegenerative and metabolic studies.
The Eye-Bank is grateful for the selfless generosity of eye donors and their families. Eye donations for research help to advance treatments and cures for blinding eye diseases, and they provide a greater understanding of other common diseases such as Alzheimer’s and diabetes.