ZeEfficacy

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From disease models to drug discovery

During the preclinical phase, it is essential to address the safety and metabolic profile of any candidate drug, but these parameters might become accessory if the drug does not display therapeutic efficacy. To address efficacy it is necessary to show if a candidate drug promotes the recovery of a pathological phenotype in a disease model. 

A disease can be modelled in vitro and in vivo. The only aspect to consider is that disease models have to display pathological phenotypes with biological translatability towards human disease biology.

Zebrafish has revealed as a powerful in vivo system for modelling human disease and discovering new drugs – several of these drugs being now in clinical phases. The comparison with the human genome shows 82% of human genes involved in disease have a zebrafish orthologue. Zebrafish embryos are transparent and develop ex utero, allowing the direct evaluation of the in vivo impact of the drugs on molecular biomarkers, cells and organs. They are easy to breed and maintain, and fertility is high: each female produces 200 eggs per mating, offering a large number of animals to test in parallel. Since the zebrafish has mammal-analogous organs, it is more useful model than alternative in vitro systems for the study of the function of genes and the effect of drugs on humans.

Zebrafish allows performing in vivo efficacy tests, fast, with accuracy and a high biological translatability.