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Enhance Drug Development with DefiniGEN’s Efficacy Screening services

Written by DefiniGEN | 2024

Our phenotypic screening services help you move quickly and efficiently from screening to identifying promising candidates by combining our expertise at generating iPSC-derived hepatocyte cell models and associated disease models, with our wide panel of bespoke bioassays. This approach provides precise, human-relevant data to help pharmaceutical companies identify promising drug candidates faster and more effectively while de-risking their pipeline.

 

The Limitations of Traditional Screening Models

 

Traditional models, such as animal testing and primary human cell models, often fail to deliver reliable human-specific responses. Animal models introduce interspecies variations, while primary human cells are prone to batch-to-batch variability and rapid functional decline. These challenges result in inconsistent data and unreliable predictions of therapeutic efficacy, which in turn lead to therapeutic candidates not passing clinical trials.

 

DefiniGEN’s iPSC-derived hepatocyte models, provide a superior alternative by offering:

 

  • Accurate Human Phenotype Recapitulation: As iPSCs are reprogrammed from human cells, they closely replicate human biology, offering predictive insights into drug efficacy.
  • Accurate Human Disease Phenotype: Leveraging the physiological relevance of our in iPSC-derived Hepatocytes, we have developed a wide portfolio of monogenic and multifactorial liver disease models that fully demonstrate the disease phenotype in vitro, including Alpha-1-Antitrypsion Deficiency (A1ATD), Wilson’s Disease, Urea Cycle Disorders, Intrahepatic Cholestasis, and Steatotic Liver Disease (MASLD).
  • No Batch-to-Batch Variation: Unlike primary human hepatocytes, iPSC-derived hepatocytes are derived from a single donor, ensuring reproducibility in your experiments.
  • Non-Cancerous Origin: Unlike immortalized cell lines, iPSC-derived hepatocytes retain normal cellular functionality and karyotype, leading to biologically relevant data.
  • Scalability for High-Throughput Investigations: Opti-HEP allows researchers to conduct high-throughput drug screening on a large scale, without compromising data integrity. This scalability accelerates compound profiling and lead optimization.

 

 

 

Comprehensive Screening using DefiniGEN’s iPSC-Derived Hepatocytes

 

Screening Services: At DefiniGEN, we aim to work as partners with your team, offering a wide range of screening services on our in vitro liver cell models, including:

  • Cell viability and cytotoxicity
  • Oxidative stress
  • Metabolite secretion assays (e.g., albumin, urea, glucose)
  • Glycogen accumulation
  • Lipid accumulation
  • Copper accumulation
  • Transporter activity
  • High-throughput protein imaging
  • mRNA expression analysis
  • And many more upon request

Bespoke iPSC-Derived Models: In addition to our pre-made liver disease models mentioned above, we can develop custom liver disease models specifically tailored to your research needs.

Applicable to Any Therapeutic Modality: We have extensively characterized our in vitro liver models and demonstrated their ability to be used as platform for the large-scale screening of small compounds as well as novel therapeutics, including:

  • GalNAc conjugates
  • RNAi/mRNA/oligonucleotides
  • Adeno-Associated Viruses (AAV)
  • DNA plasmids
  • Lentiviral vectors
  • Many other modalities upon request

 

Ready to enhance your drug screening capabilities with iPSC-derived models and high-throughput scalability? You can learn more about our Efficacy screening services here, or you can contact one of our experts today.