Applied StemCell Inc. (ASC)
Leading Gene & Cell Therapy CRO/CDMO
Advancing Gene and Cell Product Development Through iPSC and Genome Editing Technologies
Applied StemCell strives to advance gene-editing and stem cell innovation for biomedical research and the biotechnology industry to assist in the development of breakthrough therapeutic approaches to cure intractable diseases.
ASC is a trailblazing CRO/CDMO committed to building a complete iPSC-Gene Editing platform for the production of iPSC-derived products for gene and cell therapy research and development. With the integration of our unique iPSC approach and IP-based site-specific large cargo insertion technology, TARGATTTM, we (1) develop immune-compatible master iPS cell lines and (2) deliver large DNA fragments to pluripotent cells that have proven differentiation capabilities. We stand at the forefront of the iPSC-based therapy revolution delivering research- and GMP-grade iPSC solutions along with various downstream assay services.
Transform the Biotechnology Industry Through the Development of Allogenic Cell Products
As an innovative company, we are committed to providing excellent iPSC services and high-quality products while working to improve and expand our capabilities to become a leading CRO/CDMO in the biotechnology industry. Our goal is to develop allogeneic cell products using the iPSC and TARGATTTM gene insertion technology platform. We hope to facilitate the creation of allogenic CAR-T and CAR-NK products with our TARGATTTM-iPSC capabilities to accelerate the manufacturing of essential therapies for cancer treatment. Furthermore, we are determined to form meaningful partnerships with companies shaping the next generation of therapeutics for regenerative medicine with our multifaceted platform to deliver life-changing iPSC-derived products.
With high-impact technologies, we offer complete service platforms and multipurpose products. ASC holds the required licensing to generate, genetically modify, and differentiate iPSCs. Additionally, we use two powerful gene-editing tools, CRISPR and TARGATTTM to engineer cell lines and animal models; generate bioproduction CHO cells and transgenic animals; and produce allogeneic products. Together, these powerful tools allow us to support the current needs of the research community, but we actively work to augment our development and manufacturing capacity to provide greater iPSC-derived product solutions for the biotechnology industry.
Induced Pluripotent Stem Cell Technologies
Complete iPSC Service Platform
Complementary Gene Editing Platform: TARGATT™ & CRISPR/Cas9
TARGATTTM - Site-Specific Knock-in Technology
We hold exclusive rights from Sandford for the groundbreaking, site-specific knock-in technology, TARGATTTM. TARGATTTM is an efficient, fast system that permits the integration of a large fragment, up to 20 kb, at a pre-selected safe-harbor locus. The single-copy insertion of any gene of interest, including chimeric antigen receptor (CAR) genes, at a transcriptionally active locus, enables the evasion of significant problems that arise from random insertion such as gene interruption. Moreover, it eliminates the position effect and guarantees high-level, uniformed transgene expression.
ASC holds rights to CRISPR/Cas9 technology and is one of the earliest providers of CRISPR services. Throughout the years we have optimized our protocols to deliver high-throughput genome editing services for complex and mainstream genetic engineering of iPSCs.
TARGATT™-Mediated Genome Engineering
Advantages vs. Other Systems
TARGATT™ iPSC-iNK Platform
ASC integrated its TARGATT™ and iPSC technology to create the TARGATT™ Master iPSC Platform (TARGATT™ iPSC-iNK Platform) for the development of off-the-shelf, allogeneic therapeutic cells, including CAR-T or CAR-NK cells, neurons, and various other cell types. The TARGATT™ Master iPSCs have a pre-engineered landing pad that enables the safe and efficient insertion of any gene of interest (GOI), such as CAR, at a safe-harbor locus. These cells can be further differentiated into any cell type. It is our goal to help address unmet medical needs through the production of CAR-T and CAR-NK therapies, and we believe that our all-encompassing TARGATT™-iPSC service platform will assist in the efforts to address issues such as the negative effects of random CAR gene insertion, the low efficiency of CRISPR, the high-cost of lentiviral CAR-T/NK manufacturing, and the need for allogenic therapeutics.