hPSC-based Regenerative Medicine by Innovative Technology Completely Overcoming Tumorigenicity

Platform technology with high through put design and development of tumorigenic cell-targeting lentiviral vector (TC-LV), and producing master cells

Advantages

➢ TC-LV technology enables high through put design, development and production of vectors with optimized promotor, marker gene and suicide gene.
➢ We proved that TC-LV completely eliminated undifferentiated or tumorigenic cells in hPSCs in vitro and in vivo even after transplantation.
➢ Genome editing contributes to safer master cell bank.

Background and Technology

Although tumorigenicity remaining after differentiation of hPSC
(human Pluripotent Stem Cells) is the most severe issue, there is no technology that overcomes the problem and eliminates hPSC-derived tumorigenic cells in vivo after transplantation.

Here, we developed TC-LV technology as innovative platform which enables high through put design, development and production of vectors with optimized promotor, marker gene (fluorescent protein; FP) and suicide gene (SG).(Fig.0)

For eliminating undifferentiated or tumorigenic cells from hPSCs, we designed a specific TC-LVs with herpes simplex virus thymidine kinase (HSV-tk) as the SG, Venus as the marker gene and survivin as the promotor (Fig.1) 1. After infection of the TC-LVs into hPSCs, undifferentiated hPSCs were sorted by Venus positive and killed by addition of nontoxic pro-drug
ganciclovir (GCV) which is phosphorylated by HSV-tk and exerts cytotoxicity (Fig.2). 1 Enriched undifferentiated hPSCs sorted by Venus positive were transplanted into mice. GCV suppressed the hPSCs-derived tumor growth completely (Fig.3).1

We further developed a method to transfect the abovementioned gene cassette into a genomic safe harbor by using gene editing technology and generate safer master cell bank. Further developments and applications on our platform will be ready for clinical use.

Reference and Patent

1. Ide et al., Stem Cells (2018) 36:230–239.
US15/111,084(pending), EP15737214.5 (pending), JP6358713 (registered), and the others

Researcher

Ken-ichiro Kosai, M. D., Ph. D. Chairman and Professor, Department of Gene Therapy and Regenerative Medicine, Kagoshima University Graduate School of Medical and Dental Science

Proposal

Worldwide exclusive license to use this technology for your regeneration medicine development.

Product No. BK-03455

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