Stem cells’ versatility in drug development  


This article is sponsored by Charles River.

Research tools, safety testing and regenerative medicines – these endless stem cell applications are powering precision, speed, and new modalities in drug development.    

Stem cells for use in drug discovery

Stem cells are fast becoming an invaluable tool in the drug discovery process. Through the use of human induced pluripotent stem cells (iPSCs) derived cells, we can improve translation and reduce risk of clinical failure. To do this successfully, it is imperative to start with quality control assays to assess routine stem cell cultures. Select mature stem cell lines can then be used in small-scale in vitro assays, and proven biochemical and phenotypic screens supporting target validation and deconvolution studies. Differentiation of these cell lines into multiple cell types/tissues, such as neurons, microglia, myocytes, organoids, and 3D models, presents opportunities to progress lead optimisation within the same systems. To ensure their efficacy, Charles River developed QC characterisation and morphological and functional assessment using High Content Imaging.   

Disease-relevant human cell models  

Developing screening, and profiling assays using disease-relevant human cell models can best mimic a given disease state and provide the earliest prediction of how a compound may translate to clinic. Our scientists have experience in a variety cell types across many disease states: 

  • Metabolism: Adipocytes, Hepatocytes, Beta Cells (pancreatic islets), Skeletal Myoblasts​, Skeletal Myotubes​, Osteoblasts​ 
  • Inflammation: Endothelial Cells​, Fibroblasts, Basophils, ​Mast Cells, Macrophages, Neutrophils, Dendritic Cells​, Keratinocytes, Chondrocytes 
  • COPD/CF/Rare Diseases: ​ Bronchial Epithelial Cells, Fibroblasts, Myoblasts, Macrophages, Neurons​
  • Neurology:  Astrocyte, Neurons, Human iPSC,hESC/Fetal Brain and rodent primary 

Sourcing neurodegenerative disease cells that are scalable and reproducible is challenging. In partnership with’s precision iPSC reprogramming, we provide access to a broadening bank of authentic human iPSC cells. This approach to cell differentiation generates high-purity, mature, consistent, and scalable human cell lines. The combination of’s human disease cells and Charles River Assay Development Services provide access to reproducible cell populations and robust assay quality. This pairing includes reprogrammed cells for both wild and mutation-specific Huntington’s Disease (H), Amyotrophic Lateral Sclerosis (ALS), and Alzheimer’s Disease (AD) and is expanding to include custom disease-relevant mutations.   

Disease relevant in vitro assays improve translation in vivo, leading to clinical success. 


Stem cells in toxicology studies

The FDA’s Modernisation Act 2.0 amends how studies can be performed with alternative animal models to access toxicity before a compound moves into clinical trials. As classical in vitro assays used in drug screening and profiling have adopted human derived stem cells for better translation, the same can be done with classical toxicity assays.  

These human stem cells can be transformed into the cells of human organs like the heart and liver. Reprogrammed iPCS also provide cell types that are often limited to toxicologists such a variety of human brain cells, a therapeutic area in desperate need of more accurate tools.   

Using reprogrammable stem cells provides a new supply chain and consistent cell lines for toxicity studies; increasing study relevance and predictability while creating vistas of opportunity for new innovative assays that can accelerate the drug development timeline.  

Stem cells as therapeutics

The use of stem cell technology as a therapeutic is an emerging field with multiple approaches, applied to many therapeutic areas such as oncology, CNS, metabolic, and immunological disorders. 

Regenerative medicine

Stem cells can be guided into becoming specific cells designed to repair or regenerate damaged cells into regaining normal function. Current trials include regeneration of neural pathways in the spinal cord and replacing damaged heart tissue after a cardiac arrest.  

Gene therapy 

In the same way that viruses replicate by injecting their genetic material into living cell, gene therapy uses viruses to insert therapeutic genes into stem cells. The use of CRISPR technologies can be used to correct inherited mutations. These modified stem cells are then be reintroduced into the patient’s body to correct gain of or loss of function.  


Immunotherapy uses one’s own body to harness the immune system and stem cells have been found to be part of the innate and adaptive immune systems. However, engineering stem cells for use in immunotherapy provides an ‘off-the-shelf’ platform and potential for more general use therapies.  

Although stem cell technology is an emerging field it is encouraging to see innovative applications from drug discovery to safety assessment, and variety of therapeutic approaches that have already seen success in the clinic.  

To learn more: Charles River Stem Cells  

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