Using our worldwide network of clinical collaborators, we access families with rare diseases of relevance to drug discovery, including families with Extreme Genetics®, a severe (extreme) version of a more common disease, families with Opposite Phenotypes presenting the mirror image features of a common disease and Genetically Modified Models.

We have developed a pipeline of novel small molecule and other therapeutic drugs against our genetically derived targets. As part of maintaining a balanced and broader pipeline, we have entered into select partnerships with top-tier pharmaceutical companies.