Applied StemCell Inc. (ASC) is a fast growing biotechnology company headquartered in Milpitas, California.
The company was founded on May 8, 2008 with a goal to advance gene-editing and stem cell innovation for biomedical research and the biotechnology industry.
After more than 11 years' of research and development, ASC is proud to offer an optimized series of tools for basic research study, drug discovery, bio-processing, bio-production and preclinical applications.
We are currently focusing on three areas:
Cell line generation for bio-production and bioassays
Patient-relevant cell models for personalized medicine and
Physiologically predictive animal models of human diseases
We are striving to improve and expand our technologies and product lines to meet the increasing requirements of the biomedical community.
- Establishing disease-specific isogenic models
- Developing master cell lines for antibody/protein production
- Generating iPSCs from patients containing the corrected mutations or inserted genes for pre-clinical applications
- Generating animal models (mouse/rat models)
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)
Our therapeutic pipeline will have a strong focus on monogenic blood disorders that are characterized by high genetic penetrance.
Such diseases are a strong point for the company, which specializes in the genetic engineering of stem cell lineages.
For patent licensing, business development, and investment inquiries:
Our Therapeutic Development Timeline:
Our genome editing TARGATTTM Alt-NHEJ has
2 to 7-fold higher gene knockin efficiency than CRISPR/HDR
Current DNA Knock-in strategy
Improved DNA Knock-in
Advantages of Our Gene Editing Therapy:
- Lifetime Effect: Therapeutic gene integrated into the genome instead of episomal
- Safe: Site--specific gene integration
- High Potency and efficact: New AAV capsids
- Lower immunogenicity: Lower viral dose
- Lower cost