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Our Science

We believe there is a treatment available for every rare genetic disease, we just need to find it!

Modelis’ approach is fully integrated from bench to bedside.  Once a lead compound is identified, we develop the capabilities to perform early stage clinical trials, taking advantage of fast-track pathways from which rare disease drug development can benefit. We offer a complete bench-to-bedside solution that extends beyond pre-clinical testing. 

Contact us to learn more about how to start your project. 

What do we do?

Modelis’ approach integrates thousands of small molecules from public and private libraries that are known to be safe. Thanks to our unique expertise, we are able to quickly and efficiently test how they can be repurposed for rare genetic diseases.

Our approach is saving time and money

Modelis’ drug discovery road reshapes the classical approach by initiating preclinical studies that will then identify new mechanisms of action. 

How do we do it and why is it unique?

From worms to humans, Modelis takes advantage of multiple, complementary animal models to encapsulate the complexity of the diseases in order to maximize cost-efficient drug discovery. Our approach leverages AI driven data processing so that we can rapidly collect, analyze, and learn from the results. 



Our unbiased phenotype-based screening approach allows identification of new mechanisms of action that would not have been thought to be involved in the studied disease via a classical, target-based approach. Modelis initiates in vivo functional assays to validate the mechanism of action of the lead candidate molecule (see below). Leveraging the genetic and pharmacological accessibility of our small model organisms, we confirm the implication of the known molecular target(s) of the hit molecule in a new context of the disease studied.


Our in vivo screening pipeline is supported by machine learning: from data analysis and interpretation, to decision making for further screening. Our artificial intelligence and medicinal chemistry partners are helping Modelis identify structure-activity relationships in order to unravel new molecules with potential therapeutic effects. 

Modelis does research differently

Our research leverages small animal models to test existing drugs to see how they effect genetic diseases. Thanks to their small size, worms and fish are central in Modelis’ process to offering a fast and cost-friendly approach for patients and partners.

We leverage simple models

Did you know that 80% of the genes causing human diseases are evolutionarily conserved in fish and worms? Humans, fish, and worms have a lot in common, which is why Modelis leverages these animals to iteratively repurpose existing treatments in a fast and cost-friendly way.

We broaden the spotlight of drug discovery

Traditional drug discovery approaches have narrowed the focus on specific therapeutic targets at a molecular level. This approach is high risk with a lower chance of breakthroughs because it may be missing the potential cure. Modelis’ approach broadens the scope of molecules tested which increases the likelihood of finding new treatments.

We are creating strategic partnerships

We are partnering with genetic sequencing centres, biotech and pharmaceutical companies to accompany families in their diagnostic journey and further expand the number of molecules available for testing. 

See our research at work

Patients become partners as we move towards finding a cure for their rare genetic disease. 

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