• 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.

Support Materials
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


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.


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


RNA-seq using NGS (next generation sequencing)

2-8 weeks


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.

Have Questions?

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