Discovery: Model Generation and Validation
ASC offers a full suite of comprehensive services tailored towards your preclinical stage cell and gene therapy (CGT) projects. Leverage our fully integrated service platform starting with in vitro model generation and cell-based assays, followed by custom animal models and in vivo assays for target validation for early go-no-go decisions of your biotherapeutics. We have a unique team of scientists with multidisciplinary expertise in CRISPR/ genome engineering technologies, stem cell biology, immuno-oncology, gene therapy models, viral vectors, and bioinformatics for every aspect of your CGT projects. We can generate in vitro and in vivo models, design viral vectors (AAV, lentivirus) and develop proof-of-concept and target validation assays specific to your therapeutic pipeline needs.
Cell Line Model Validation
Animal Model Validation
Cell and Gene Therapy has progressed very rapidly with the advent of new technologies for genome editing, stem cell and immuno-oncology applications. Applied StemCell (ASC) has been at the forefront of these technologies for >11 years and has been a leading CRO service provider for discovery phase of our customer’s cellular and gene therapy projects. Using CRISPR/cas9 and our proprietary TARGATT™ transgene integration technologies, we have generated 1300+ custom cell line and animal models for target discovery, disease modeling and drug screening applications.
We now leverage our extensive experience in cell and animal biology to develop clinically relevant in vitro and in vivo models and downstream validation of these models for more advanced cellular and gene therapy pipelines.
Cell and gene therapy drugs have a unique mechanism of action. Unlike traditional small molecule drugs, detailed characterization of CGT involves development of custom assays specific to the unique mechanism of action of the drug. Due to target specificity of these drugs, traditional animal/ cell line models may not work. ASC specializes in developing the ideal research model for your biotherapeutic that would enable you to have better translation of your results in clinical setting.
Have an idea but need the resources to develop your cell and gene therapy product? We can help!
- Derive/ differentiate stem cell lines
- Genetically engineer cell lines for immunotherapy applications, cellular therapy
- Design and generate gene therapy constructs
- Screen for tissue-specific promoters
- Viral vector serotypes analysis (for AAV and lentiviral)-based delivery of gene therapy products,
- Characterize the mechanism of action of your drug candidate.
We also provide expert consultation and downstream assay development and IND-enabling studies to advance your biotherapeutic projects.
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- Baskfield, A., Li, R., Beers, J., Zou, J., Liu, C., & Zheng, W. (2019). An induced pluripotent stem cell line (TRNDi009-C) from a Niemann-Pick disease type A patient carrying a heterozygous p. L302P (c. 905 T> C) mutation in the SMPD1 gene. Stem cell research, 38, 101461.
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