Abstract
Presentation Description :
In 2003 we set out to explore the possibility of using human embryonic stem cells (hESCs) as a platform for the treatment of retinal disease. In this presentation I will describe our journey from the development of a directed differentiation protocol that allows efficient derivation of retinal pigment epithelium (RPE) cells from hESCs "in the culture dish" to the application of this technology in an ongoing phase I/IIa trial in patients with advanced dry age-related macular degeneration. Milestones along the way that are relevant to the process of drug / treatment development include in-vitro characterization of cell differentiation, testing in animal models, the transition from basic research to the translational phase that included the production of clinical grade hESC-derived RPE cells under xeno-free conditions, rigorous testing to ensure safety and efficacy of these cells, designing a phase I/IIa clinical trial, obtaining FDA approval and finally launching of the clinical trial. Lessons learned along the way will be shared.
This is an abstract that was submitted for the 2018 ARVO Annual Meeting, held in Honolulu, Hawaii, April 29 - May 3, 2018.