Safety and stable survival of stem-cell-derived retinal organoid for 2 years in patients with retinitis pigmentosa.

Transplantation of induced pluripotent stem cell (iPSC)-derived retinal organoids into retinal disease animal models has yielded promising results, and several clinical trials on iPSC-derived retinal pigment epithelial cell transplantation have confirmed its safety. In this study, we performed allogeneic iPSC-derived retinal organoid sheet transplantation in two subjects with advanced retinitis pigmentosa (jRCTa050200027). The primary endpoint was the survival and safety of the transplanted retinal organoid sheets in the first year post-transplantation. The secondary endpoints were the safety of the transplantation procedure and visual function evaluation. The grafts survived in a stable condition for 2 years, and the retinal thickness increased at the transplant site without serious adverse events in both subjects. Changes in visual function were less progressive than those of the untreated eye during the follow-up. Allogeneic iPSC-derived retinal organoid sheet transplantation is a potential therapeutic approach, and the treatment's safety and efficacy for visual function should be investigated further.
Competing Interests: Declaration of interests Y.H. reports that Kobe City Eye Hospital received a research grant from the Japan Agency for Medical Research and Development (AMED) (grant number: JP19bk0104082) and financial support from Sumitomo Pharma Co. Ltd. for this study. S.S., T.M., M.Y., and M.T. are employed by Vision Care, Inc. T.H., A.N., K.U., K.O., H.O., M. Fujiwara, S. Yamasaki, K.W., K.B., K.K., A.I., and A.K. are employed by Sumitomo Pharma Co. Ltd. T.K. is a board member of Sumitomo Pharma Co. Ltd. The authors are co-inventors on patent applications.
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