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- Retinal sheet (DSP-3077)
Retinitis pigmentosa (RP) is a group of inherited retinal disease caused by the loss or dysfunction of light-responsive photoreceptors or retinal pigment epithelial cells. RP first results in loss of night and peripheral vision but ultimately affects central vision in most cases. RP is genetically heterogenous with causative mutations identified in over 70 genes to date. Although the progression of RP is highly variable among individuals, common pathology includes progressive degeneration (cell death) of rod photoreceptors (cells responsible for scotopic vision) followed by that of cone photoreceptors (cells responsible for photopic vision, visual resolution, and color vision). After a long period of progression, RP often leads to severe visual impairment. RP is the second leading cause of blindness in Japan, therefore the development of new treatments for this serious condition is highly desired.
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Current Treatments
A standard treatment to restore lost function of the retina or halt disease progression has not been developed yet. The use of oral vitamin A palmitate to delay disease progression has historically been advised by many specialists but with controversial efficacy. Although gene therapies have been studied, they can only be used if the RP-causing gene has been identified and if sufficient viable target cells remain for a therapeutic effect. Retinal sheets processed from human embryonic tissues have been administered to restore lost photoreceptors, and potential efficacy findings have been reported in human clinical studies. There are, however, ethical and logistical concerns with the use of human retinal sheets derived from embryonic donors.
Our Goal with Regenerative
and Cellular Therapy
The technology used for DSP-3077 is based on the self-organizing cell culture technique, named SFEBq method, an efficient differentiation method from pluripotent stem cells into three dimensional neural tissues originally developed by Dr. Yoshiki Sasai’s research group at RIKEN. We have established a manufacturing process to generate induced pluripotent stem cell (iPSC)-derived retinal sheet in collaboration with RIKEN and Sumitomo Chemical Co., Ltd. An iPSC-derived retinal sheet has the potential to engraft into a severely degenerating retina and the grafted sheet gives rise to functionally matured photoreceptors. These photoreceptors can form connections with host bipolar cells resulting in restoration of visual function.
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Regenerative Medicine for Cell Replacement Using Organoids
- Transplant DSP-3077 into the subretinal space
- Connect iPSC-derived photoreceptors with the patient’s bipolar cells to restore function