Induced pluripotent stem cell derived retinal organoid sheets for regenerative therapy in patients with end-stage retinitis pigmentosa

Abstract

Purpose : Retinitis pigmentosa is a hereditary retinal degenerative disease characterized by night blindness and progressive loss of visual field due to the degeneration of the retinal rod photoreceptor cells. Induced pluripotent stem cells (iPSC)-derived retinal organoid sheet showed a potential function after transplantation in animal models of retinal degereration. In this clinical study, allogenic iPSC-retinal organoid sheets were transplanted in the eye of 2 patients with end-stage rtinitis pigmentosa to study the safety and survival of the graft,

Methods : The primary endpoint was the survival and safety of the transplanted retinal organoid sheets in the first year after transplantation, and the secondary endpoints were the safety of the retinal sheet transplantation procedure and visual function evaluation. The first patient was a 67-year-old woman with a corrected visual acuity of hand motion who recieved three retinal organoid sheets near the macula of the right eye; the second patient was a 42-year-old man with a corrected visual acuity of 0.05 who recived three retinal organoid sheets near the macula of the right eye. The patients were followed-up for one year.

Results : The grafts survived in a stable condition in both patients through the first year after transplantation with the increased retinal thickness at the transplant site. No serious adverse events were observed in either case. The first case showed a partial improvement in visual function tests including full-field stimulus testing and Table Test.

Conclusions : Allogeneic iPSC-derived retinal organoid sheet transplantation is a safe and practical therapeutic approach. The treatment efficacy in terms of visual function should be further investigated.

This abstract was presented at the 2023 ARVO Annual Meeting, held in New Orleans, LA, April 23-27, 2023.