Event Title : Retinal cell replacement in Macular Degeneration: Bench to Bedside
Loss of retinal pigment epithelial (RPE) cells results in progressive, irreversible blindness in dry age-related macular degeneration (AMD). Dry AMD is the largest cause of incurable blindness for people over 50 and it is estimated that over 1.5-4 Cr people suffer from this disease in India alone. Transplantation of RPE cells derived from human induced pluripotent stem cells (hiPSCs) offers an attractive therapeutic prospect for patients globally. Some attempts to transplant hiPSC-RPE cells in patients suffering from dry AMD have demonstrated safety and suggested the potential efficacy of this strategy. At Eyestem, we have successfully generated retinal pigment epithelial (RPE) from clinical grade hiPSCs employing a novel GMP compliant protocol and performed in depth characterization of the differentiated cells with respect to identity, purity and potency. We then tested the safety and efficacy in RCS rat model of retinal degeneration post sub-retinal transplantation of Eyecyte-RPE. We have further had an in-depth pre-IND meeting with the FDA to understand the regulatory path to taking this therapy to patients. I will share our experience from this unique and challenging journey of taking India’s first pluripotent stem cell-based product from the bench to the clinic.