Retinal pigment epithelial cell manufacturing technique ready for Phase I clinical trials

Written by RegMedNet

A collaborative team from the National Institute of Health and the National Eye Institute (both MD, USA) has developed a novel GMP compliant manufacturing process for iPSC-derived retinal pigment epithelial cells, as a potential treatment opportunity for age-related macular degeneration.

As the leading cause of vision loss in the elderly, age-related macular degeneration (AMD) invites investigation into preventative and restorative treatment strategies. At present, no cure exists for the ‘dry’ or atrophic of AMD, but a combined team from the National Institute of Health and the National Eye Institute (both MD, USA) may have developed a revolutionary technique for retinal pigment epithelial (RPE) cell regeneration. 

The validated, GMP compliant manufacturing process was presented at the American Society of Gene & Cell Therapy Annual Meeting (29 April—2 May 2019; DC, USA) and utilizes an open manufacturing system, including integrated quality measurements and criteria at multiple stages. 

The process begins with deriving iPSCs from CD34 cells harvested from the patient as a starting material, which are differentiated utilizing a defined mixture of cytokines and reagents before iPSC-specific marker loss and RPE-specific marker gain are assessed. 

Following RPE differentiation and maturation, the process involves seeding the cells onto a PLGA scaffold-snapwell plate to be cultured for an additional month, before the scaffold containing the RPE cells is transplanted into the retinal surface of the patients’ eye.

The team predicts that patients may be enrolled and treatment administered as part of Phase I clinical trials as early as the first quarter of 2019. 

Source: Sarkar S, Thakkar A, Hua F. Development and Validation of a GMP Process to Generate Retinal Pigment Epithelial Cells (RPE) Derived from Autologous Induced Pluripotent Stem Cells (iPSCs). Presented at: American Society of Gene & Cell Therapy Annual Meeting. Washington, DC, USA. 2019