iPSC Disease Modeling - Drug Screening

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Drug discovery and development for neurological disorders has lagged far-behind therapeutics for other disease areas. One of the main reasons is due to the high failure rate of drug candidates due to lack of predictive in vitro and in vivo models, problems in sourcing reliable models, complexity in molecular mechanisms and disease pathologies that have been hard to elucidate due again to the lack of biorelevant models for basic research.

The ground-breaking technological advancements in the past decade(s) in bioinformatics, genome editing, stem cell technologies, and targeted drug discovery has revitalized drug discovery and development for neurological disorders. Importantly, the iPSCs and highly efficient protocols for directed-differentiation to neuronal lineage cells have had a major impact in this renewed interest in CNS therapeutics by providing a consistent, reproducible, and non-invasive resource for sourcing patient-specific cells and generating biorelevant models. As well, with the CRISPR genome editing capabilities of iPSCs, any number of in vitro models of neurological disorders and isogenic control lines can be engineered very easily for reliable experimentation.   

As a leader in stem cell and genome editing technologies, Applied StemCell has leveraged our extensive expertise in these technologies to provide an efficient in vitro platform for screening your pharmaceutical and biological drug candidates for efficacy before moving onto costly animal tests.

We can generate iPSCs from your patient samples and use optimized directed-differentiation protocols to generate functional neurons and glial cells for screening your compounds.

We also have panels of “ready-to-use” iPSCs and differentiated cells that have been reliably tested using known neurotoxins and neuroprotective agents:

Avoid late-stage drug attrition! Our battery of quantitative and qualitative assays allow you to reliably assess the efficacy your early-stage drug candidates using iPSC-derived cells before moving onto nonclinical in vivo studies: We provide stage-specific phenotype assays in our drug 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


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

Can’t see an assay you are looking for? Don’t be discouraged. Please contact us with your requirements.