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Safety

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

Once a drug candidate has been identified and developed, safety and toxicity testing are critical next steps to ensure that no unexpected immune-related adverse events (IrAEs), such as cytokine release syndrome, are induced upon drug treatment.

Drug safety, also known as pharmacovigilance, involves assessing a drug's potential to cause harm. The aim is to identify, understand, and minimize adverse effects before the drug enters human trials. Selecting the appropriate preclinical model is crucial for ensuring that your results are translatable and mimic a human response.

If you have finished in vitro testing of your lead compound and are now seeking the most suitable in vivo preclinical model to evaluate your construct’s safety, genOway is here to support your next steps.

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

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Define your target and scientific questions​

Having comprehensive background information is essential for selecting the right model for your research needs. Additionally, by discussing with us your questions, such as which cells or organs you believe might be affected the most, or feedback loops that might be activated, it allows us to give you better-informed guidance. You can contact us and talk with ourscientific experts — we will be happy to discuss your data to ensure that weprovide you with the most appropriate model for your research.

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

  • Are there known or predicted off-targets that the drug may bind to? Is there potential for cross-reactivity with healthy cells/tissues?
  • Do you know the pathways in which your drug target is involved? Are there known feedback loops?
  • Are there known safety biomarkers that can be monitored in your preclinical studies?
  • Are anti-drug antibodies (ADAs) likely to affect your drug’s safety

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Find the right model for your research​

Relying on wild-type mice or inappropriate humanized models may result in a potentially viable drug candidate being rejected, or important adverse drug reactions not being detected, as occurred in the TeGenero 2006 clinical trial with TGN1412. This can happen when drug safety experiments are performed in PBMC-reconstituted mouse models that do not consider the contribution of myeloid cells to the elicited immune response and which contain a majority of pre-activated T cells.

We have developed a wide range of translational pre-clinical models to meet your research needs, which can be used alone or in complementary ways to maximize and accelerate your research. They are either readily available for purchase (see our Catalog Models) or can be developed specifically for you (see our Customized Models).

Catalog Models: rodents and cell lines

Our selection of catalog models includes different types of models useful for safety assessment.​

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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. These models are engrafted with human CD34+ hematopoietic stem cells, which provide a much more complete reconstitution of the immune system compared to human PBMCs, thereby making them more translational to clinical studies. Given their functional human myeloid and lymphoid compartments, permissiveness to PDX and CDX engraftments, and extended lifespan, these models are ideal for safety assessment of immunotherapies targeting myeloid and lymphoid cells, as shown in articles published by both Sensei Bio and genOway.

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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-hCTLA-4 and the genO-hCD47/hSIRPα models, among many others. With these models you can assess the safety of your human-specific immunomodulators/immunotherapies. These models have proved successful in contributing to the safety testing of new therapies, as shown by Maxivax  and Caedo Oncology.
  • Humanized CD3 and CD28 models are also available to help you assess the safety of new T-cell engagers, such as the genO-panhCD3 and genO-hCD28 models, as presented at the SITC 2022[MR3] , and AACR 2024[MR4] , respective[FS5] ly.
  • The humanized TFRC model (hCD71) allows you to test the safety of constructs aiming to cross the blood-brain barrier, or therapies targeting CD71-overexpressing cancers, as demonstrated by Bratti et al., 2024[MR6] .
  • Our humanized FcγR model is a powerful tool for safety assessment of Fc-competent therapeutic antibodies.

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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. 

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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. 

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.

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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.

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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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Other applications

Efficacy

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

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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