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:
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.
Using our TARGATT™ technology, a gene-of-interest can be specifically inserted at a well-characterized, transcriptionally active locus in the genome with guaranteed transgene expression.
This site-specific Knock-in technology has been first established in mouse models, and we have successfully applied it to human cell lines.
We are moving forward to various projects that will combine the CRISPR/Cas9 technology and TARGATT™ for highly specified transgene expression.
TARGATT™ Technology Applications:
- SITE-SPECIFIC INTEGRATION OF TRANSGENES (Patent Pending)
- NOVEL INTEGRATION SITES AND USES (Patent Pending)
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.
Leverage Applied StemCell’s TARGATT™ Technology for your Gene and Cell Therapy Projects:
- Up-to-date strategies: Construct design and optimization
- Safe integration: Site-specific at a well-defined intergenic locus
- High potency and efficacy: New AAV capsids
- Lower immunogenicity: Lower viral dose
- Lower cost