Table 1 Summary of preclinical rat and pig studies performed to demonstrate safety and efficacy of clinical-grade AMD iPSC-RPE patch.

PR, photoreceptor; WT, wild-type; na, not available.

PhaseGroupNo. of eyesDuration
1iPSC-RPE patch safety study* in RNU (Crl:NIH-Foxn1rnu) rats (0.5-mm-diameter iPSC-RPE patch)
1aSham surgery1070/8 STEM121 positive (2 unscheduled deaths)
1biPSC-RPE sheet2076/15 eyes showed integrated iPSC-RPE patch (one
unscheduled death)
2Cell suspension safety study* in RNU (Crl:NIH-Foxn1rnu) rats (100,000 cells)
2aVehicle control550/5 STEM121 positive
2bPure iPSCs1053/10 teratoma
2cScaffold pieces cut from 8-mm2 PLGA punch1050/10 STEM121 positive
2diPSC-RPE cell suspension1051/10 endo-ophthalmitis; 9/10 STEM121 and PMEL17 positive;
9/10 Ki67 negative; no teratoma
3Cell suspension and patch efficacy study in RCS rats (1 mm iPSC-RPE patch or 100,000 cells)
3aSham surgery185 /10PR degeneration
3bSuspension (100,000 cells)115 /10PR rescue in all eyes
3cSheet (1-mm scaffold)75 /108/10 eyes showed integrated iPSC-RPE patch and PR rescue
4Empty PLGA scaffold safety study in WT pig eye (4 × 2 mm PLGA piece)
4aSham surgery310na
4bEmpty PLGA scaffold310No inflammation, slow degradation of PLGA, and recovery of
mfERG signals
5iPSC-RPE patch efficacy study in laser-injured pig eye
5aEmpty PLGA scaffold (4 × 2 mm)38Slow degeneration of PRs
5biPSC-RPE patch (4 × 2 mm)38Integration of iPSC-RPE patch in pig eye and no degeneration of PRs
5ciPSC-RPE cell suspension (100,000 cells)38
5diPSC-RPE transwell (4 × 2 mm)38No degeneration of PRs, but no integration of iPSC-RPE
transwell in pig eye

*Body weight and food consumption were checked weekly for immunocompromised rats in the safety study. There was no apparent effect of the patch or cell suspension on weight and food consumption of these animals.