Newsletter
Drug Toxicity and Efficacy Testing
Make Informed Go-No-Go Decisions Early in Your Drug Development Process!
Leverage Applied StemCell’s unique expertise in CRISPR/Cas9 and induced pluripotent stem cell (iPSC) technologies to engineer predictive and reliable in vitro disease models as an alternative to animal models for preliminary drug screening.
Our ISO:9001 certified service platform offers full flexibility in choosing assay modules with a wide range of functional endpoints for early-stage in vitro screening of preclinical drug candidates. We have a comprehensive cell-based test battery from which you can choose assays for efficacy, safety or target discovery that suit your therapeutic pipeline. We provide the scientific expertise, accurate and efficient screening to help you make informed decisions about your small/ large molecules early in your drug development process.
Human CD34+ cells, NK cells, T cells & more human primary cells are now available!
Use our high-quality primary cells to compare your genetically modified iPSC differentiated cells. Our scientists can help you develop the right assays to collect the comparative data you need to carry your research forward. Contact us to schedule your free consultation today.
Read about our NK Cytotoxicity Assay Service and our successful case study comparing human iPSC-derived NK cell function to human primary NK cells.
Products and Services
Technical Details
Physiologically relevant cell line models and cell-based in vitro assays are becoming crucial tools for screening new drug candidates before moving to expensive testing using animal model. The development of cutting-edge new technologies such as CRISPR/Cas9 and induced pluripotent stem cell (iPSC) technologies has enabled the engineering of predictive and reliable in vitro disease models which afford an inexpensive, faster, efficient, and ethically compatible alternative to using costly, animal models for preliminary drug screening.
1. In Vitro Disease ModelingGenome Editing/ Correction iPSC Reprogramming & Differentiation |
2. Drug ScreeningPhenotypic Screening Functional Validation Cell Viability/ Toxicity Mitochondrial/ Hepatic/Cardio Toxicity Customized Assay Development |
1. In Vitro Disease Modeling:
Screen your compounds in a wide-variety of cell lines! Applied StemCell is a market leader in gene editing technologies. We have engineered > 500 distinct cell lines with a wide variety of mutations, using a combination of different technologies (CRISPR/Cas9, TARGATT™ and more) and techniques (transfection/transduction) to generate many different cell line models that are perfectly suited to screen candidate compounds in your therapeutic pipeline.
- Cancer cells and cell line gene editing
- iPSC/ ESC gene editing
- Primary cell line models
- 3D cell line/ iPSC models
We also provide comprehensive iPSC services and products for iPSC-based neurotoxicity & efficacy screening:
- iPSC generation
- iPSC differentiation into tissue lineages
- iPSC and differentiated cell lines and other products
2. Drug Screening:
Flexible/ customizable project options to suit your screening needs! Below is a sample list of some of the assays we offer.
Screening |
Types of Assays |
Estimated Timeline |
Cytotoxicity & Cell Viability Assays |
MTT/ MTS cell proliferation assay LDH, Necrosis and Apoptosis assays Luciferase (bioluminescence) expression cAMP level measurement |
4-6 weeks |
Mitochondrial Toxicity Testing |
Enzyme activity Volume fraction detection |
2-4 weeks |
Functional Assays |
Calcium influx/ imaging Electrophysiology: Multielectrode array (MEA) analysis and Patch clamp recording |
8-12 weeks |
Quantitative Gene Expression |
qPCR RNA-seq using NGS (next generation sequencing) |
2-8 weeks |
Morphology |
Neurite growth assay Biomarker screening |
2-4 weeks |
Custom Assays |
iPSC generation; characterization; gene editing; differentiation Custom assay development |
Based on project requirements |
Our custom screening service is ideal for CNS, cardiovascular, metabolic, cancer, and immunotherapy drug candidates.
Don’t see an assay you want? CONTACT US with your project requirements.
Support Materials
Publications
Cell and Gene Therapy Applications:
- Chen, H., Shi, M., Gilam, A., Zheng, Q., Zhang, Y., Afrikanova, I., ... & Chen-Tsai, R. Y. (2019). Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII. Scientific reports, 9(1), 1-15.
Other CRO Services:
- Mace, E. M., Paust, S., Conte, M. I., Baxley, R. M., Schmit, M., Mukherjee, M., ... & Akdemir, Z. C. (2019). Human NK cell deficiency as a result of biallelic mutations in MCM10. bioRxiv, 825554.
- Baskfield, A., Li, R., Beers, J., Zou, J., Liu, C., & Zheng, W. (2019). Generation of an induced pluripotent stem cell line (TRNDi004-I) from a Niemann-Pick disease type B patient carrying a heterozygous mutation of p. L43_A44delLA in the SMPD1 gene. Stem cell research, 37, 101436.
- Hong, J., Xu, M., Li, R., Cheng, Y. S., Kouznetsova, J., Beers, J., ... & Zheng, W. (2019). Generation of an induced pluripotent stem cell line (TRNDi008-A) from a Hunter syndrome patient carrying a hemizygous 208insC mutation in the IDS gene. Stem cell research, 37, 101451.
- Cheng, Y. S., Li, R., Baskfield, A., Beers, J., Zou, J., Liu, C., & Zheng, W. (2019). A human induced pluripotent stem cell line (TRNDi007-B) from an infantile onset Pompe patient carrying p. R854X mutation in the GAA gene. Stem cell research, 37, 101435.
- Yang, S., Cheng, Y. S., Li, R., Pradhan, M., Hong, J., Beers, J., ... & Zheng, W. (2019). An induced pluripotent stem cell line (TRNDi010-C) from a patient carrying a homozygous p. R401X mutation in the NGLY1 gene. Stem cell research, 39, 101496.
- 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.
- Huang, W., Xu, M., Li, R., Baskfield, A., Kouznetsova, J., Beers, J., ... & Zheng, W. (2019). An induced pluripotent stem cell line (TRNDi006-A) from a MPS IIIB patient carrying homozygous mutation of p. Glu153Lys in the NAGLU gene. Stem Cell Research, 101427.
- Sundararaj, K. P., Rodgers, J., Angel, P., Wolf, B., & Nowling, T. K. (2020). Neuraminidase activity mediates IL-6 production through TLR4 and p38/ERK MAPK signaling in MRL/lpr mesangial cells. bioRxiv.
- Li, R., Baskfield, A., Lin, Y., Beers, J., Zou, J., Liu, C., ... & Zheng, W. (2019). Generation of an induced pluripotent stem cell line (TRNDi003-A) from a Noonan syndrome with multiple lentigines (NSML) patient carrying a p. Q510P mutation in the PTPN11 gene. Stem cell research, 34, 101374.
- Li, R., Pradhan, M., Xu, M., Baskfield, A., Farkhondeh, A., Cheng, Y. S., ... & Rodems, S. (2018). Generation of an induced pluripotent stem cell line (TRNDi002-B) from a patient carrying compound heterozygous p. Q208X and p. G310G mutations in the NGLY1 gene. Stem Cell Research, 101362.
- Poli, M. C., Ebstein, F., Nicholas, S. K., de Guzman, M. M., Forbes, L. R., Chinn, I. K., ... & Coban-Akdemir, Z. H. (2018). Heterozygous Truncating Variants in POMP Escape Nonsense-Mediated Decay and Cause a Unique Immune Dysregulatory Syndrome. The American Journal of Human Genetics, 102, 1-17. https://doi.org/10.1016/j.ajhg.2018.04.010
Vozdek, R., Long, Y., & Ma, D. K. (2018). The receptor tyrosine kinase HIR-1 coordinates HIF-independent responses to hypoxia and extracellular matrix injury. Sci. Signal., 11(550), eaat0138.