GMP Grade iPSC Services & Products
Applied StemCell (ASC) is in the process of getting GLP and GMP certified and complied with. With its core technologies, CRISPR, TARGATTTM, and iPSC, ASC can produce almost any gene modification including therapeutic gene/reporter gene insertion, point mutation correction, small nucleotide addition, replacement, and deletion.
ASC can now offer a wide range of GMP clinical-grade induced pluripotent stem cell (iPSC) related services. Applied StemCell’s GMP Grade iPSC line (Cord Blood CD34+, Male) can be engineered to express almost any gene of interest (CRISPR; TARGATTTM), but ASC can also conduct gene-editing services with the iPSC lines provided by customers. GMP grade knockin cell line generation service using the TARGATT™ Master iPSC Line is also available for single-copy gene insertion at a safe harbor locus.
Along with gene-editing services, ASC offers comprehensive iPSC differentiation service to committed somatic lineages. ASC can work with its GMP iPSC lines or researchers can send in their healthy, diseased, or gene-edited iPSC lines and Applied StemCell will produce the iPSC differentiated cells in its GMP facility.
ASC is working towards developing a comprehensive inventory of GMP clinical-grade iPSC-related products and is currently building a TARGATTTM Universal Modifiable iPSC platform for therapeutic cell production.
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.
iPSC Expansion and Banking
ASC provides iPSC expansion and banking of its cGMP iPSCs. We also provides custom iPSC generation services using an epsiomal reprogramming method.
Licensing Information: No license, no passthrough royalty
iPSC Gene Editing
With 12+ years of stem cell & genome editing experience, ASC offers high-quality gene editing services using the latest technology, CRISPR and TARGATTTM.
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
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.
Stocking of Frozen Cells
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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.
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).