AbstractCell based transplantation aimed at the replacement of lost dopamine (DA) neurons holds great potential for the treatment of Parkinson’s disease (PD). Considerable progress has been made in generating fully functional and transplantable dopamine (DA) neurons from human pluripotent stem cells (hPSCs) and the field is rapidly developing. We have developed robust and efficient differentiation protocols resulting in the formation of authentic and functional DA neurons from human embryonic stem cells (hESCs). Here, I will summarize the translational trajectory and describe the GMP manufacturing, pre-clinical validation and regulatory process embarked on to bring this hESC-derived dopamine cell product to clinical trial for PD.
Embryonic stem cells, dopamine neurons, Parkinson’s Disease