iPSC quality control standardization
exploratoryresearch program · medium · Thu Jan 01 2026 00:00:00 GMT+0000 (Coordinated Universal Time)
Support harmonized quality control for induced pluripotent stem cell-derived therapies, interoperable iPSC banks, regulatory alignment, and scalable manufacturing of regenerative therapies.
International Quality Assessment Rounds comparing flow cytometry-based undifferentiated-state assessment and qPCR-based genomic integrity testing across laboratories, including standardized workflows and marker performance evaluation.
2026 Stem Cell Reports publication reported multi-site QAR 2019 and QAR 2023 findings and identified OCT3/4, TRA-1-60, and SSEA5 as robust pluripotency-associated markers.
QAR 2019 showed high consistency in genomic integrity testing but substantial variability in flow cytometry; QAR 2023 standardized workflows enabled systematic cross-site marker evaluation and produced empirical evidence for reproducible iPSC QC.
PSC-derived therapy translational best practices
exploratoryresearch program · medium · Thu Jan 01 2026 00:00:00 GMT+0000 (Coordinated Universal Time)
Define best practices and decision points for translating pluripotent stem cell-derived products from laboratory research into licensed therapies.
ISSCR best-practices resource synthesizing scientific, technical, and regulatory guidance for development of PSC-derived therapies.
2026 Stem Cell Reports publication described the ISSCR Best Practices for the Development of Pluripotent Stem Cell-Derived Therapies resource.
The resource provides global regulatory perspective, development-process guidance, and links to jurisdictional regulatory information for PSC-derived therapy translation.
bemdaneprocel
phase 3drug program · high · Tue Jun 17 2025 00:00:00 GMT+0000 (Coordinated Universal Time)
Replace lost or damaged dopaminergic neurons in adults with Parkinson's disease to restore dopaminergic function and improve or stabilize clinical outcomes.
Cryopreserved, off-the-shelf human embryonic stem cell-derived dopaminergic neuron progenitor cell therapy grafted bilaterally into the putamen; Phase 3 randomized, sham surgery-controlled, double-blind efficacy and safety study.
Phase 3 study BRT-DA01-301 in approximately 102 adults with Parkinson's disease was published in ClinicalTrials.gov on 2025-06-17.
Phase I trial met primary safety and tolerability objectives at one year after transplantation, reported no adverse events related to the cell product, showed increased putaminal 18F-DOPA PET uptake at 18 months indicating graft survival, and reported improvement or stability in secondary and exploratory clinical outcomes, including average 23-point MDS-UPDRS Part III OFF improvement in the high-dose cohort.
OpCT-001
phase 1drug program · high · Mon Mar 10 2025 00:00:00 GMT+0000 (Coordinated Universal Time)
Evaluate a cell therapy approach for adults with primary photoreceptor disease, with emphasis on safety, tolerability, visual function, functional vision, and anatomic measures of engraftment.
Phase 1/2a first-in-human, multisite, two-part interventional study; Phase 1 uses dose escalation and Phase 2 gathers additional safety and clinical outcome data across subgroups.
CLARICO study OpCT-001-101 in approximately 54 adults with primary photoreceptor disease was published in ClinicalTrials.gov on 2025-03-10.
Parkinson's disease diary assessment study
exploratoryresearch program · medium · Fri Jul 29 2022 00:00:00 GMT+0000 (Coordinated Universal Time)
Evaluate how assessment frequency affects variability over time, reliability, and compliance for Parkinson's disease diary use in patients whose medications do not adequately control symptoms.
Noninterventional study evaluating Parkinson's disease diary use and assessment frequency.
ClinicalTrials.gov record for the noninterventional Parkinson's disease diary study was published on 2022-07-29.
