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Series GSE229768 Query DataSets for GSE229768
Status Public on Sep 25, 2023
Title Preclinical quality, safety and efficacy of a human embryonic stem cell-derived product for treatment of people with moderate Parkinson’s Disease (STEM-PD) III
Organism Homo sapiens
Experiment type Genome variation profiling by SNP array
SNP genotyping by SNP array
Summary Cell replacement therapies for Parkinson’s Disease (PD) based on transplantation of dopaminergic neurons generated from pluripotent stem cell sources are now entering clinical trials.  Here, we present the quality, safety and efficacy data supporting a first-in-human clinical trial in PD using an embryonic stem cell product STEM-PD, as well as the design of the trial itself. The cryopreserved STEM-PD product was manufactured under Good Manufacturing Practice (GMP) and fully quality-tested in vitro for regulatory compliance. The product was further tested in an extensive 39-week Good Laboratory Practice (GLP) safety study in immunodeficient rats for toxicity, tumourigenicity and biodistribution, as well as in a 24-week non-GLP efficacy study. These studies showed that the transplanted STEM-PD cells could induce full functional recovery in a pre-clinical rat model of PD, and that the treatment did not give rise to any adverse effects. This cell batch will be used for all participants in the STEM-PD Phase I/IIa clinical trial, where the first of 8 patients with moderate PD have now been transplanted. Furthermore, we observed highly comparable in vivo efficacy results between two different GMP batches of STEM-PD cells, verifying that this product has the potential to be serially manufactured for widespread clinical use. 
Overall design gDNA from a stem cells (RC17) master cell bank (MCB) and from two different ventral midbrain dopaminergic precursors (STEM-PD) batches (#1 and #3) was purified and analysed using the HumanCytoSNP-12 v2.1. The genetic stability of the MCB and of STEM-PD #1 was tested at thaw and after different passages in culture.
Contributor(s) Graziano S, Pikkupeura S
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Submission date Apr 14, 2023
Last update date Sep 25, 2023
Contact name Malin Parmar
Organization name Wallenberg Neuroscience Center, MultiPark and Lund Stem Cell Centre
Department Department of Experimental Medical Science, Lund University
Street address 221 84 Lund
City Lund
ZIP/Postal code Box 117, SE-221 00
Country Sweden
Platforms (1)
GPL13829 Illumina HumanCytoSNP-12 v2.1 BeadChip
Samples (6)
GSM7177499 RC17 master cell bank at thaw
GSM7177500 RC17 master cell bank two passages after thawing
GSM7177501 RC17 master cell bank four passages after thawing
This SubSeries is part of SuperSeries:
GSE229769 Preclinical quality, safety and efficacy of a human embryonic stem cell-derived product for treatment of people with moderate Parkinson’s Disease (STEM-PD)
BioProject PRJNA955800

Download family Format
SOFT formatted family file(s) SOFTHelp
MINiML formatted family file(s) MINiMLHelp
Series Matrix File(s) TXTHelp

Supplementary file Size Download File type/resource
GSE229768_MCB_matrix.csv.gz 11.8 Mb (ftp)(http) CSV
GSE229768_MCBp2_matrix.csv.gz 44.8 Mb (ftp)(http) CSV
GSE229768_MCBp4_matrix.csv.gz 44.8 Mb (ftp)(http) CSV
GSE229768_RAW.tar 23.9 Mb (http)(custom) TAR (of IDAT)
GSE229768_STEMPD1_matrix.csv.gz 11.8 Mb (ftp)(http) CSV
GSE229768_STEMPD1p14_matrix.csv.gz 11.8 Mb (ftp)(http) CSV
GSE229768_STEMPD3_matrix.csv.gz 11.8 Mb (ftp)(http) CSV
Processed data are available on Series record

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