Long-Term Efficacy of GMP Grade Xeno-Free hESC-Derived RPE Cells Following Transplantation

McGill T.J., Bohana-Kashtan O., Stoddard J.W., Andrews M.D., Pandit N., Rosenberg-Belmaker L.R., Wiser O., Matzrafi L., Banin E., Reubinoff B., Netzer N., Irving C.

Trans Vis Sci Tech., 2017

Publication from researchers at Cell Cure Neurosciences where they display the efficacy of RPE cells derived under xeno-free conditions from clinical and xeno-free grade human embryonic stem cells following transplantation into the subretinal space of Royal College of Surgeons (RCS) rats. BioLamina’s laminin cell culture substrate is being used in the differentiation protocol. The results of this study demonstrate that the transplantation of OpRegen into the subretinal space of RCS rats protected the retinal structure, rescued visual function, preserved rod and cone photoreceptors long-term (up to 180 days). Transplanted RPE cells were identified in both the subretinal space and integrated into the host RPE monolayer in animals of all age groups, and often contained internalized photoreceptor outer segments. Optomotor tracking was rescued in a dose-dependent manner. The outer nuclear layer was significantly thicker in cell-treated eyes than controls up to P150. No pathology was observed. This data combined with data collected in definitive safety studies (tumorigenicity and spiking and safety/biodistribution) has resulted in an FDA approved IND and a Phase 1/2a clinical trial for AMD patients is ongoing, NCT02286089.