iPSC platform

An industry leading iPSC-based drug discovery platform  

Patient-derived induced pluripotent stem cells (iPSCs) provide unique opportunities for phenotypic screening to identify new mechanisms of disease. Evotec has built an industrialised iPSC infrastructure that represents one of the largest and most sophisticated platforms in the industry. Evotec's iPSC platform has been developed over the last six years with the goal to industrialise iPSC-based drug screening in terms of throughput, reproducibility and robustness to reach highest industrial standards. This effort was initially enabled by a research collaboration with Harvard University involving world-leading scientists at the Harvard Stem Cell Institute. More recently, Evotec established collaborations with Celgene, Sanofi, Center for Regenerative Therapies Dresden, Censo Biotechnologies and Fraunhofer IME-SP to support Evotec's growing iPSC activities. In addition, we are working together with a number of academic institutions to further broaden and strengthen our iPSC capabilities.

Creating "patients in a dish"

Modelling human disease in a dish and high-throughput screening offers greatest therapeutic potential, however, technical challenges exist in culturing and differentiating these iPSC-derived cells at large scales. Evotec has overcome such hurdles through generation of a cell bank of fully validated iPSC lines, successful scale-up of the manufacturing process and strict quality control parameters.


XLII cell::explorer system

Strong focus on standardisation, scaling and QC is essential for iPSC-based drug discovery:
  •  High-quality iPSC cultures at large scale (QC)
  • Industrial processes for the generation of patient-derived target cell types
  • Robust disease-relevant phenotypic readouts for screening
  • Panel of patient-derived genotypes for patient stratification


Fully integrated iPSC-based drug discovery platform established - from iPSCs to drug candidates and beyond

iPSC culture
Patient-derived disease models
Hit identification
Hit-to-lead/ Lead-optimisation
In vivo pharmacology
Access to iPSC lines
SC-derived disease relevant cell types
High-content screening
Cellular assays
Expansion & banking
Medicinal chemistry
Biomarker-driven in vivo disease models
Quality control standards

Gene expression profiling

Target deconvolution
In vitro toxicity
iPSC culture Access to iPSC lines
Patient-derived disease models SC-derived disease relevant cell types
Hit identification High-content screening
Hit-to-lead/ Lead-optimisation Cellular assays
In vivo pharmacology PK/PD
iPSC culture Expansion & banking
Patient-derived disease models Optimisation
Hit identification Electrophysiology
Hit-to-lead/ Lead-optimisation Medicinal chemistry
In vivo pharmacology Biomarker-driven in vivo disease models
iPSC culture Quality control standards
Patient-derived disease models Scale-up
Hit identification

Gene expression profiling

Hit-to-lead/ Lead-optimisation Target deconvolution
In vivo pharmacology Biomarkers
iPSC culture Scale-up
Patient-derived disease models Automation
Hit identification
Hit-to-lead/ Lead-optimisation In vitro toxicity
In vivo pharmacology


iPSC technology started a new era for drug discovery providing human disease model systems, in which multiple, biologically relevant targets and pathways are simultaneously interrogated. 

Evotec’s iPSC platform is closely connected to a variety of in-house key technologies, defining disease signatures and causal relationships between genetic mutations and perturbation of specific molecular pathways. This creates powerful human translational models that will ultimately enable us to identify better targets and drugs for those patients suffering from diseases with high unmet medical need.

Evotec is running multiple drug discovery programmes using human iPSCs:

Neurodegenerative diseases
Lysosomal storage diseases
Neurodevelopmental diseases
Alzheimer`s disease 
Gaucher disease
Fragile X syndrome
Age-related macular degeneration
Autosomal dominant polycystic kidney disease (ADPKD)
Amyotrophic lateral sclerosis
Niemann Pick disease type C 
& others
& others
Diabetic nephropathy
Frontotemporal dementia
Mucolipidosis type IV 
Chronic kidney disease (CKD)
Huntington`s disease
Batten’s disease
Parkinson`s disease
Neurodegenerative diseases Alzheimer`s disease 
Lysosomal storage diseases Gaucher disease
Neurodevelopmental diseases
Fragile X syndrome
Age-related macular degeneration
Nephropathies Autosomal dominant polycystic kidney disease (ADPKD)
Neurodegenerative diseases Amyotrophic lateral sclerosis
Lysosomal storage diseases Niemann Pick disease type C 
Neurodevelopmental diseases & others
Retinopathies & others
Nephropathies Diabetic nephropathy
Neurodegenerative diseases Frontotemporal dementia
Lysosomal storage diseases Mucolipidosis type IV 
Neurodevelopmental diseases
Nephropathies Chronic kidney disease (CKD)
Neurodegenerative diseases Huntington`s disease
Lysosomal storage diseases Batten’s disease
Neurodevelopmental diseases
Neurodegenerative diseases
Parkinson`s disease
Lysosomal storage diseases
Neurodevelopmental diseases

Further drug discovery programmes in other disease areas are currently being set up.

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Our iPSC-based drug discovery alliance with Celgene DDup # 8 - iPSC-based drug discovery Our in vitro iPSC research servcies

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