Applied StemCell Presentation: Jump Start Allogenic Cell Therapies using cGMP-Grade TARGATT Master iPSCs
Thursday, May 30, 2024 06:00 PM - 06:15 PM
Exhibit Hall - Theatre B
Global Showcase Presentation
Jump Start Allogenic Cell Therapies using cGMP-Grade TARGATT Master iPSCs
Presenter
• Ruby Tsai, CEO, Applied StemCell, USA
Induced pluripotent cells (iPSCs) for site-specific DNA-fragment knock-in are essential for developing and manufacturing the next generation of cell therapeutic products. Traditional genome editing technologies, such as CRISPR/Cas9 systems, can be used to establish stable knock-in cell lines; however, transgene size can be limited, and substantial licensing fees are required to move the engineered cell line into product development and manufacturing.
To generate a ready-to-use cGMPgrade iPSC line for site-specific gene of interest (GOI) knock-in, we adapted the TARGATT system by integrating an attB landing pad into the Hipp11 locus (H11) by MAD7 in a cGMP-grade iPSC line, and isolated desired clones harboring the landing pad, which then were confirmed by Sanger Sequencing. With an established cGMP-grade TARGATT iPSC line, we then inserted a single copy of RFP construct at the H11 locus to verify efficiency of knock-in (insertion) as well as expression robustness and the ability of engineered iPSCs to be differentiated into natural killer (NK) cells (iPSC-derived NK cells).
Our data demonstrates that the TARGATT system can be particularly useful for cancer immunotherapy applications. For example, unique therapeutic genes (such as CAR constructs) can be inserted in the cGMP TARGATT Master iPSC line for differentiating into NK cells or T cells that could potentially be utilized to fight various serious human diseases.
Session Objectives:
• Platforms for iPSC Gene Editing (Allogenic)
• Analyze the merits and demerits of employing existing methods in iPSC gene editing platforms, emphasizing the inventive strategy employed by Applied StemCells.
• Investigate the prospective medical uses of cutting-edge gene-editing and iPSC technologies, including iPSC-CAR-NK.
• Assess the role of iPSC allogeneic platforms in various medical contexts, including regenerative medicine, immune oncology, and cell therapeutics.
Presenter
• Ruby Tsai, CEO, Applied StemCell, USA
Induced pluripotent cells (iPSCs) for site-specific DNA-fragment knock-in are essential for developing and manufacturing the next generation of cell therapeutic products. Traditional genome editing technologies, such as CRISPR/Cas9 systems, can be used to establish stable knock-in cell lines; however, transgene size can be limited, and substantial licensing fees are required to move the engineered cell line into product development and manufacturing.
To generate a ready-to-use cGMPgrade iPSC line for site-specific gene of interest (GOI) knock-in, we adapted the TARGATT system by integrating an attB landing pad into the Hipp11 locus (H11) by MAD7 in a cGMP-grade iPSC line, and isolated desired clones harboring the landing pad, which then were confirmed by Sanger Sequencing. With an established cGMP-grade TARGATT iPSC line, we then inserted a single copy of RFP construct at the H11 locus to verify efficiency of knock-in (insertion) as well as expression robustness and the ability of engineered iPSCs to be differentiated into natural killer (NK) cells (iPSC-derived NK cells).
Our data demonstrates that the TARGATT system can be particularly useful for cancer immunotherapy applications. For example, unique therapeutic genes (such as CAR constructs) can be inserted in the cGMP TARGATT Master iPSC line for differentiating into NK cells or T cells that could potentially be utilized to fight various serious human diseases.
Session Objectives:
• Platforms for iPSC Gene Editing (Allogenic)
• Analyze the merits and demerits of employing existing methods in iPSC gene editing platforms, emphasizing the inventive strategy employed by Applied StemCells.
• Investigate the prospective medical uses of cutting-edge gene-editing and iPSC technologies, including iPSC-CAR-NK.
• Assess the role of iPSC allogeneic platforms in various medical contexts, including regenerative medicine, immune oncology, and cell therapeutics.
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