• Drug Toxicity and Efficacy Testing

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.

 

FAQ

 

Support Materials
Application Notes

 

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 Modeling

Genome Editing/ Correction
 (Cancer cells, iPSCs, ESCs, primary cells)

iPSC Reprogramming & Differentiation

3D Cell/ iPSC Culture

button-gray-forward-arrow 

2. Drug Screening

Phenotypic 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.

 We also provide comprehensive iPSC services and products for iPSC-based neurotoxicity & efficacy screening:

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.

Case Studies

 

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 reports9(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 research37, 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 research37, 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 research37, 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 research39, 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 research38, 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 research34, 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, 1-17https://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.

Have Questions?

An Applied StemCell technical expert is happy to help, contact us today!

Google