NIH Research Festival
Age-related macular degeneration (AMD) is the leading cause of vision loss in the elderly people and no cure exists for the "dry" or atrophic form to date. Approximately 11 million people are affected in the U.S alone and, with the rise in the aging population, this number is predicted to increase significantly. The mechanism of vision loss is mainly due to atrophy of retinal pigment epithelium (RPE) cells, which leads to the death of surrounding photoreceptors. It has been shown in animal models that RPE transplantation can prevent photoreceptor loss and therefore, prevent disease progression of AMD. We have developed a GMP compliant manufacturing process to differentiate iPSC into RPE cells to be used in a phase I clinical trial for patients with AMD. In our process, iPSC derived from patient CD34 cells are used as starting material (see Hua et al. for manufacturing of iPSC). iPSC are carefully differentiated using a defined cocktail of cytokines and reagents and are assessed for the loss of iPSC-specific markers and the gain of RPE-specific markers outlined here. After RPE differentiation and maturation, cells are seeded onto a PLGA scaffold-snapwell plate and cultured for an additional month. The scaffold containing RPE cells is transplanted into retinal surface of the patients’ eye. The validated manufacturing process outlined herein utilizes an open manufacturing system with quality measurements and criteria built in at multiple stages. We predict that this phase I clinical trial will be open and enrolling patients toward the end of 2018.
Scientific Focus Area: Stem Cell Biology
This page was last updated on Friday, March 26, 2021