Review Article

Cell Therapy for Retinal Dystrophies: From Cell Suspension Formulation to Complex Retinal Tissue Bioengineering

Table 1

Current or planned clinical trials based on hPSCs to treat RP, Stargardt’s macular dystrophy (SMD), or AMD.

Stage of developmentTargeted diseaseSponsor/companyTherapy

Phases I and II
NCT02563782, NCT01469832, NCT01344993, NCT01345006
AMD, SMDAstellas Institute for Regenerative MedicineAllogenic hESC-RPE, cell suspension injection
Phase I/II
NCT01674829
Dry AMDCHA Biotech Co. Ltd.Allogeneic hESC-RPE, cell suspension injection
Phase I/II
NCT02286089
Dry AMD, GACell Cure Neurosciences Ltd.Allogeneic hESC-RPE, cell suspension injection
Phase I/II
NCT02590692
Dry AMD, GARegenerative Patch Technologies LLCAllogeneic hESC-RPE, epithelium on a parylene membrane
Phase I/IIAMDRIKEN Centre for Developmental BiologyAutologous and allogenic hiPSC-RPE, epithelium without substrate
Phase I
NCT01691261
Wet AMDPfizer/London Project to Cure BlindnessAllogenic hESC-RPE, epithelium on polyester membrane
cGMP optimizationAMD, SMD, RPCellular Dynamics International/NEIAutologous hiPSC-RPE, epithelium on biodegradable scaffold
Phase I/IIRP with genetic defects in LRAT, MERTK, RPE65I-Stem/Institut de la VisionAllogeneic hESC-RPE, epithelium on amniotic membrane
PreclinicalAMD, best disease, LCAHMC, IsraelAllogeneic hESC-RPE, cell suspension injection
Phase I/II
NCT03046407, NCT02749734, NCT02755428
Dry AMD, SMDChinese Academy of Sciences, Southwest Hospital, ChinaAllogeneic hESC-RPE
Phase I/II
NCT02903576
Dry AMD, wet AMD, SMDFederal University of São PauloAllogeneic hESC-RPE as cell suspension versus as a sheet on a polymeric substrate