iPSC Disease Modeling – Drug Screening

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:

• Control “master” iPSC lines that can be used as parental cells for genome engineering relevant cell line or disease model, and further differentiated into neurons and glial cells
• Isogenic control neurons (cortical, dopamine) and astrocytes differentiated from our control, master iPSC line (ASE-9109 and ASE-9110)
• Isogenic panel of iPSCs engineered with neuronal gene knockout that can be differentiated into neuronal lineage cells and have been shown to recapitulate neurological disease phenotypes
• Reporter iPSC lines that can be differentiated to neuronal lineage of choice and provide a research tool for quantitative safety assessments of chemicals.

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:

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