Newsletter
GMP Grade iPSC Services & Products
Applied StemCell (ASC) is GLP and GMP certified. With our core iPSC and genome-editing technologies (CRISPR & TARGATT™), we can generate, genetically modify, and differentiate iPS cells. We use an episomal method to generate iPSCs from a variety of tissue samples (e.g., PBMCs, fibroblast, etc.). The reprogrammed pluripotent cells or our GMP Grade iPSC Line (Cord Blood CD34+, Male) can be engineered to express almost any gene of interest using CRISPR or TARGATT™. With TARGATT™, we can deliver a single copy of a large DNA fragment at a transcriptionally active safe harbor locus that contains a landing pad. Our team of experts can properly handle the genetically altered iPSCs and further differentiate the cells into committed somatic lineages, such as NK and T cells.
In an effort to expand our service offerings and product catalog, our team is developing a comprehensive inventory of GMP clinical-grade iPSC-related products and currently building a TARGATT™ Universal Modifiable iPSC platform for therapeutic cell production.
- cGMP iPSC Expansion & Banking; Master Cell Bank (MCB); Working Cell Bank (WCB)
- Customized iPSC Generation (Episomal Method)
- iPSC Gene Editing: CRISPR/Cas9 Gene Editing
- iPSC Gene Editing: TARGATT™ Gene Editing
- In-Process QC (Reagent QC (plasmid map/sequence), Validation (cell, gRNA, integrase), and more)
- iPSC Differentiation: Customer Provide Protocol
- iPSC Differentiation: ASC’s SOP
- Cell Banking & Storage with 24/7 Monitoring System
ASC’s cGMP Grade iPSC Line
Applied StemCell’s cGMP Grade iPSC Line was generated from male, CD34+ cord blood using an episomal reprogramming method. Master cell bank or working cell bank are available.
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Applied StemCell’s cGMP Grade iPSC Line |
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Source cell type: CD34+ Cord Blood, male |
Reprogramming method: Episomal |
Master cell bank or working cell bank available |
Product QC:
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iPSC Expansion and Banking
ASC provides iPSC expansion and banking of its cGMP iPSCs. We also provide custom iPSC generation services using an epsiomal reprogramming method.
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cGMP iPSC Expansion and Banking |
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Number of Vials |
Cell Count |
Other Deliverables |
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Master Cell Bank |
50-200 vials |
1x10^6 cells/vial |
QC and CoA |
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Working Cell Bank |
50-200 vials |
1x10^6 cells/vial |
QC and CoA |
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Customized iPSC Generation (Episomal Method) |
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Number of Clones |
Number of Vials |
Cell Count |
Other Deliverables |
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Seed Bank |
3 Clones |
10 vials |
1x10^6 cells/vial |
QC Characterization and CoA |
iPSC Gene Editing
With 13+ years of stem cell & genome editing experience, ASC offers high-quality gene editing services using the latest technology, CRISPR and TARGATTTM.
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iPSC Gene Editing |
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Service |
Details |
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CRISPR/Cas9 Gene Editing |
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TARGATT™ Gene Editing |
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GMP-like Plasmid Production For Gene Editing |
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In-Process QC:
- Reagent QC (plasmid map/sequence)
- Validation (cell, gRNA, integrase)
- Transfection pool genotyping
- Clone genotyping
TARGATTTM Site-Specific Knockin Technology
Unlike other gene editing companies, ASC has a unique gene-editing technology, TARGATTTM, that enables site-specific, irreversible gene insertion at a safe-harbor locus, H11 or ASC2. The unidirectional gene insertion at an active, intergenic locus does not disrupt other internal genes. TARGATTTM works well in non-dividing cells and permits single-copy insertion of large fragments up to 20kb. ASC has observed high integration efficiencies and medium to high protein expression in stable cells.
The NIH awarded Applied StemCell with a $2M SBIR grant for the improvement of its hTARGATTTM technology for human gene and cell therapy. According to the NIH ASC’s proprietary gene editing technology
“could efficiently and safely treat more than ten thousand monogenic human diseases.”
- NIH grant review group summary statement
TARGATTTM Universal Modifiable iPSC Platform
TARGATTTM Master iPSCs
With TRAGATTTM technology the possibilities are endless! Applied StemCell plans to build a modifiable master iPSC line using donor iPSCs, pluripotent stem cells that have been engineered to evade immune recognition. The master iPSC line will carry a pre-engineered landing pad in a safe harbor-locus for fast and efficient gene knockin by TARAGTTTM integrase.
ASC’s Universal iPSC Line:
- Genomic safe harbor locus for gene insertion (ASC IP)
- High-efficiency integration (ASC IP)
- Fast and simple
- Stable single-copy gene insertion (ASC IP)
- Uniform population after integration
- Immune evasive (ASC IP)
- Clinical grade iPSC
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TARGATT™ Universal Modifiable |
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Genomic safe harbor locus for gene insertion (ASC IP) |
GMP iPSC Differentiation
Applied StemCell has used its integration-free protocols to differentiate iPSCs to various committed somatic lineages, including T cells, natural killer cells, retinal pigment epithelium cells, and astrocytes. With the new GMP facility, ASC can provide these services while following good manufacturing practices (GMP) using its TARGATTTM Universal Modifiable iPSCs or the iPSC line provided by a customer.
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iPSC Differentiation |
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Service |
Details |
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iPSC Differentiation: Customer provides protocol |
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iPSC Differentiation: ASC’s SOP |
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Stocking of Frozen Cells
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Cell Banking & Storage |
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Service |
Details |
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Annual Storgae |
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Products and Services
Technical Details
GMP Facility
ASC's GMP facility is set for iPSC generation, expansion, gene editing, and differentiation as well as general cell banking processes. ASC’s GMP facility includes manufacturing space, the storage for raw and finished products, and support lab areas. ASC’s GMP facility operates under the guidelines established by the CFR (Code of Federal Regulations) Title 21, Parts 1271 (Current Good Manufacturing for Human Cells, Tissues, and Cellular and Tissue-Based Products).
Application Notes
Applications in Cell Therapy
The off-the-shelf master iPSCs have the potential to revolutionize immunotherapy. The engineered human induced pluripotent stem cells with a landing pad at a genomic safe-harbor locus can be used to insert a therapeutic gene of interest to generate an iPSCs that carry a single copy of the gene needed for therapeutic studies.
Universal Modifiable iPSC in CAR-NK Therapy
Since ASC’s Master iPSC line holds a TARGATTTM site at a safe-harbor locus, a single copy of a gene of interest such as CAR can be inserted into the genome. ASC could generate an iPSC line with a specific CAR, and use its integration-free method to differentiate the iPSCs with CAR to a committed cell type such as natural killer (NK) cells. With the rise in CAR-NK and CAR-T therapies, ASC’s TARGATTTM and iPSC differentiation capabilities offer an alternative, effective method for the development of novel CAR-NK cell therapies.