Genomic and transcriptomic quality control for an autologous iPSC-derived cell therapy for Parkinson’s disease

Bratt-Leal, Andres M., Gorba, Thorsten, Williams, Roy M., Mossman, Jim A., Hills, Rachel, Bridge, Charlotte, Lane, Emma L. and Lelos, Mariah J. ORCID: https://orcid.org/0000-0001-7102-055X 2026. Genomic and transcriptomic quality control for an autologous iPSC-derived cell therapy for Parkinson’s disease. Cell Stem Cell 33 (10) , pp. 1610-1624. 10.1016/j.stem.2026.08.014

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Abstract

Toward development of an autologous, induced pluripotent stem cell (iPSC)-based cell therapy for Parkinson’s disease (PD), we demonstrate successful, reproducible genomic and transcriptomic qualification of patient-derived dopaminergic neuron precursor cells (DANPCs) across multiple donors. Our analysis includes whole-genome sequencing data from fibroblasts, iPSCs, and DANPCs and the development of NeuriTest, an RNAseq-based bioinformatic analysis of DANPCs designed to predict cell quality based on empirical animal data. Autologous cell therapies are immune matched to the patient, potentially augmenting durability of benefit compared to allogeneic cells while negating the need for immunosuppression and accompanying side effects. Patient-specific iPSCs are an autologous cell source that can be differentiated to dopaminergic neurons, the cell type lost in PD. We report here our preclinical manufacturing strategy and results demonstrating efficacy in a PD rodent model and safety in a 9-month GLP toxicology study.

Item Type:	Article
Date Type:	Publication
Status:	Published
Schools:	Schools > Biosciences
Additional Information:	Full authors available at https://doi.org/10.1016/j.stem.2026.08.014
Publisher:	Elsevier
ISSN:	1934-5909
Date of First Compliant Deposit:	28 September 2026
Date of Acceptance:	26 August 2026
Last Modified:	03 Nov 2026 11:12
URI:	https://orca.cardiff.ac.uk/id/eprint/189823

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