Table 1.
Bioprocess characteristics and intervention outcomes for clinical trials involving pluripotent stem-cell-derived therapeutics.
| ID number | Derived cell type | Bioprocess characteristics | Manufacturing timeline | Population characterization strategies | Clinical outcomes/trial status |
|---|---|---|---|---|---|
| NCT01625559 | hESC-derived retinal pigment epithelium (Allogeneic) | hESC on mouse-feeder in static 2D culture, differentiation strategy not disclosed, in-process monitoring not disclosed | Not disclosed | Morphology, cell counts, pigmentation, karyotyping (g-banding), FACS (hPSC/hRPE markers), cell function assays, short tandem repeat analysis, sterility testing, mycoplasma testing, endotoxin analysis, GMP compliance | Phase I complete, results deemed favorable87 |
| NCT02923375 | iPSC-mesanchymoangioblast-derived mesenchymal stem cells (allogeneic) | Cymerus platform (2D system) in serum-free, feeder-free conditions, in-process monitoring not disclosed93 | Not disclosed | mRNA qPCR, antibody staining flow cytometry, comparative genomic hybridization, single-nucleotide polymorphism analysis, global transcriptome analysis, GMP-compliant manufacturing | Phase I complete. Short-term safety verified. Confounding factors obscured potential effectiveness89 |
| NCT02057900 | hESC-derived cardiovascular progenitors (allogeneic) | hESC on mouse-feeder in static 2D culture, treatment of cultures with BMP2 + SU5402 medium, downstream filtration of SSEA-1+ cells, in-process monitoring not disclosed | Not disclosed | Morphology, cell counts, marker staining, microscopic imaging, FACS (hESC/cardiac progenitor markers), HLA phenotyping, GMP compliance | Phase I complete, short-term safety verified, slightly improved symptomatic results94 |
| JPRNUMIN000011929 | iPSC-derived retinal pigment epithelium sheets (autologous) | 2D hiPSC culture, differentiation to hiPSC-RPE, seeding onto collagen type I gel, formation of monolayer, in-process monitoring not disclosed | 6 weeks to RPE sheet formation from hiPSC-RPE | Morphology, pigmentation, RT-PCR for RPE markers, assays for growth factors (ELISA), microarray genomics, MHC histocompatibility testing, DNA methylation testing, copy-number variation analysis, GMP compliance95 | Trial halted after 2 patients. Deemed ineffective, with safety concerns88 |
| NCT03482050 | ESC-derived astrocytes (allogeneic) | Not disclosed | Not disclosed | Not disclosed | Trial complete, no results posted |
| NCT03119636 | ESC-derived neural precursor cells (NPCs) (allogeneic) | EB neural differentiation strategy, not effectively disclosed for pre-clinical human ESC-derived NPCs in primates96 | 3 weeks to early neural rosette formation | Not disclosed | Unknown status |