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Efficacy

Efficacy testing is a critical step in drug development, assessing whether a drug candidate achieves its intended effect at various doses.​

Efficacy testing is a critical step in drug development, assessing whether a drug candidate achieves its intended effect at various doses. To perform a reliable and robust preclinical analysis of your drug’s efficacy, you need translational mouse models, which generate data that predicts what happens in humans. 

If you have completed the in vitro testing of your lead compound and are looking for the ideal in vivo preclinical model to analyze the efficacy of your construct, genOway can help you with the next steps.  

The importance of in vivo efficacy assessment

Performing in vivo preclinical drug efficacy tests is essential to validate your drug candidate and collect key data for IND approval. This step can help you: 

  • validate the mechanism of action of your drug in a disease-relevant model 
  • discard inefficient drugs
  • optimize drug dosage to achieve the minimal effective dose
  • understand the ideal therapeutic window and administration route

4 steps you need to follow for successful efficacy assessment of your compound

I -

Define your target and scientific questions​

Having comprehensive background information is essential for selecting the right model for your research needs. Understanding your drug target, its expression profile, and the mechanism of action — along with the associated signaling pathway — allows us to recommend the most suitable model aligned with your needs. Additionally, by discussing with us your questions, such as in which cells your target protein is expressed, and whether the expression is constant, it will allow us to give you better-informed guidance. You can contact us and talk with our scientific experts — we are happy to discuss your data to ensure that we provide you with the most appropriate model for your research.

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To help you choose the best model for your efficacy test, we may ask you some of the following questions:​

  • Does your drug bind to multiple targets?  
  • Do you know the expression profile of your drug target(s)? 
  • Do you know of protein ligands that could compete for binding and attenuate the response? 
  • Which readout (tumor volume, decrease of a specific type of cells, survival, biochemical markers, behavioral changes) do you need to assess your drug’s efficacy?

II -

Find the right model for your research​

Selecting the right preclinical model is essential for a successful transition to clinical trials. It ensures your results are relevant to human biology and helps avoid costly delays. Inappropriate model choices—such as using wild-type or mismatched humanized mice—can lead to the premature rejection of promising drug candidates. For instance, choosing mice that do not express the correct levels of your drug target in the right cells could lead to the under-representation of a drug’s efficacy. 

Finding the right model is essential to help you move seamlessly to clinical trials, ensuring that your results are translatable to humans, and saving your time. 

We offer a diverse portfolio of highly translational preclinical models designed to support your research goals. These models can be used independently or in combination to enhance and accelerate your studies. They are available off the shelf through our Catalog Models, or can be custom developed to meet your specific requirements through our Customized Models.

Catalog Models: rodents and cell lines

Our selection of catalog models includes different types of models useful for efficacy assays.​

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Target-specific humanized models​

A wide range of target-specific humanized strains is available and ready for purchase. These models are immunocompetent and display a physiological expression of the human target. Some of the models present in our catalog include: 

  • Several humanized immune checkpoint models, such as the genO-hPD-1 and the genO-hVISTA models, among many others. With these models you can assess the efficacy of your human-specific immunomodulators/immunotherapies or test their tumor growth inhibition action. These models have proved successful in contributing to the development of new therapies and advancing them to clinical trials, as shown by Anaveon and Sensei Bio. 
  • Humanized CD3 models are also available to help you assess new T-cell engagers, such as the genO-panCD3 and genO-CD3ε models. These models were validated by our clients for efficacy testing in the context of autoimmunity (Perico et al., 2024) and by Pfizer for oncology (Abayasiriwardana et al., 2025) 
  • The humanized TFRC model (hCD71) allows you to test the efficacy of constructs aiming to cross the blood-brain barrier, or therapies targeting CD71-overexpressing cancers, as presented at the AACR 2024. 
  • Our humanized FcγR model is a powerful tool for efficacy assessment of Fc-competent therapeutic antibodies, such as for the depletion of B cells or vaccinating against influenza A, as presented at the AACR 2024. 

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Cell lines

Our humanized mouse models can be used for syngeneic studies, for which there is available a complementary catalog of cell lines. 

  • A variety of cell lines are available, such as colon carcinoma cells lines (MC38 and CT26, for C57BL/6 and BALB/c mice, respectively) or a lymphoma cell line (EL4). 
  • These cell lines are engineered to express human proteins such as CD19 or CD20 (in the EL4 background), or humanized immune checkpoints (in MC38 and CT26 backgrounds). 
  • We also provide a range of reporter and Knockout cell lines, which can be used as a control for your efficacy studies and for target validation. 

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Humanized immune system (HIS) models 

As part of our catalog, we also provide humanized mouse models with a human immune system (HIS), such as the genO-BRGSF-HIS and genO-BRGSF-A2-HIS models. Given their extended lifespan, functional human myeloid and lymphoid compartments, and permissiveness to PDX and CDX engraftments, these models are ideal for efficacy assessment of immunotherapies targeting myeloid and lymphoid cells, as shown in the poster presented by Macomics and published by IGM Biosciences. 

 

Immunodeficient models 

In our vast portfolio you can also find immunodeficient rodents. If you are testing cell therapies such as CAR-T cells, or if you want to test new compounds for chemotherapy, then our immunodeficient models can be useful to you. They support PDX and CDX engraftment and can develop metastasis, as well as allow longer experimental timeframes due to their extended longevity. 

Customized Models

If you are looking for a unique model that is not part of our portfolio, we can also make a customized model for you. Our scientists will consider all of your needs and create the best model to fit your research. You can easily book a discussion session and we will guide you through the process.

III -

Design your experiments – Scientific Advice & Support​

Our team of scientific experts is here to support you in defining, designing, and optimizing your experiments to ensure the best possible outcomes. You’ll be able to discuss your project with our experienced researchers, who can collaborate with you on your study design, data interpretation, and strategic planning — before, during, and after project execution. We view ourselves as active partners in your research, committed to helping your project succeed. 

You can contact us at any moment during the process — we are here to help and willingly dedicate time to you and to your needs.

IV -

Perform your experiments at genOway or at a trusted CRO

If you lack the time or facilities to conduct your first proof-of-concept studies, we can discuss whether they can be carried out at genOway using our preclinical platform or by a Contract Research Organization (CRO). Depending on your scientific needs and on the models used, we can assist you in selecting a trusted CRO to conduct your experiments, enabling you to concentrate on advancing your discoveries.

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Safety

Safety testing is a critical step in drug development, assessing whether a drug candidate is safe.​

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Scientific excellence

From model design to experimental results
Featured in 600+ scientific articles

Collaborative approach

Collaboration with 17 Top Pharmas,
170+ Biotechs and 380+ Academic Institutions

Robust validation data on catalog models

Generated with biopharma partners and in-house

Innovative technologies

and guaranteed freedom to operate

Easy access to models

Models with certified health status from professional breeders in US and Europe