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About Us

Applied StemCell Inc. (ASC) is a fast growing Gene & Cell Therapy CRO headquartered in California, USA

Applied StemCell Inc. (ASC) is a leading Gene & Cell Therapy CRO company founded on May 8, 2008 and is headquartered in Milpitas, California. Leveraging its more than 12 years of research and development, ASC is proud to support early stage gene and cell therapy development processes with two high impact platforms: CRISPR-TARGATT™ genome editing and stem cell technologies. We are also a member of the National Institute of Technology and Standards (NIST) Genome Editing Consortium where we provide benchmark materials using our unique expertise in stem cell technology and genetic engineering to establish measurements and standards for characterizing genome editing outputs using stem cells.

 

We are currently focusing on four areas:

Integrated Stem Cell Solutions for Cell Therapy and Regenerative Medicine Research

Stem cell-based services and products to build next generation cell line models and to advance cell-based therapies

Physiologically predictive animal model generation and downstream in vivo assays for model validation and drug screening

Gene expression technology in cell lines for library screens, bioproduction and bioassays

Pre-IND enabling Gene and Cell therapy BioServices CRO with service from model generation to efficacy/toxicology studies, potency assay, pre-IND filing and CMC consultation

We strive to improve and expand our technologies and product lines to stay up-to-date with the progress in biomedical science and technology, and to meet the increasing requirements of the research community.

(1) An advanced series of services and products in stem cell technologies (iPSC/ ESCs, patient/ genome edited lines, differentiated cells, genome edited lines) to build patient relevant cell line models for allogenic banking, disease modeling, drug screening, personalized medicine, regenerative medicine, and tissue engineering.

(2) In vivo model generation (humanized and conditional/inducible expression mice/ rat) to build physiologically predictive models; and model validation and drug discovery/screening (antibody, vaccines, gene/cell therapy) using in vivo-based assays such as proof-of-concept studies, dose ranging, biodistribution, toxicity assessments for your early stage drug candidates.

(3) Innovative mammalian cell gene expression technology, TARGATT™ to build large cell-based screening libraries for protein evolution, antibody discovery and screening, and bioproduction/ bioprocessing.

(4) A comprehensive BioService CRO platform offering custom solutions for early-stage cell and gene therapy projects by leveraging our genome editing and stem cell technology expertise: AAV serotype selection, gene therapy construct design and optimization, assay development (AAV potency assays, efficacy validation, and toxicology studies); and, consultation for pre-IND filing and CMC processes.

 

Applied StemCell’s proprietary TARGATT™ technology enables site-specific integration of a gene-of-interest at a well-characterized,transcriptionally active safe harbor locus in the genome with high efficiency and high level transgene expression.

This site-specific knockin technology was first established in mouse models and has been successfully applied to human cell lines (HEK293, CHO, iPSCs), rat, pig, and rabbit models.

We offer a curated list of products and a broad range of services using TARGATT™ for: site-specific gene insertion, high level gene expression,controlled gene expression models, and more. Our TARGATT™ models are available with “docking sites” in well-defined loci such as the Rosa26, H11, and ASC’s proprietary high expression locus (ASC2). We can also custom engineer models to implement this technology for your specific purpose: custom loci, custom cell and animal models.

As well, ASC’s research and development teams are on a hard pursuit to evolve the TARGATT™ technology for ex vivo and in vivo therapeutic gene editing applications in humans (hTARGATT™) to develop safer and more effective gene and cell therapies.

Patents:

  • SITE-SPECIFIC INTEGRATION OF TRANSGENES
  • NOVEL INTEGRATION SITES AND USES
  • METHODS FOR SCREENING VARIANT OF TARGET GENE

 

TARGATT™ Technology Applications:

  • In vivo and in vitro large transgene knockin cell and animal model
  • Protein/ antibody screening libraries, bioproduction and bioprocessing
  • Cell-based therapy

 

Leverage Applied StemCell’s TARGATT™ Technology for your
Gene and Cell Therapy Projects:

Up-to-date strategies: Construct
design and optimization
Lower immunogenicity: Lower
viral dose
High potency and efficacy: New
AAV capsids
Lower cost
Safe integration: Site-specific at
a well-defined intergenic locus

Applied StemCell’s technology and scientific experience has enabled a successful gene therapy pipeline for intractable monogenic disorders, and the formation of our sister company, ASC Therapeutics (ASCTx) under the Applied StemCell, Inc corporate umbrella. Under the leadership of Dr. Ruby Chen-Tsai who spearheaded the former therapeutics program, Applied StemCell’s mission as a gene and cell therapy CRO will benefit the advancement of safer and effective gene and cell therapy programs with collaborators in the biotechnology industry.

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