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Hunt for key genes could help identify new medicines

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    UCB is working with top universities to uncover critical biological mechanisms which could lead to new treatment offerings for patients.

    In collaboration with academic partners, our scientists are exploring the genetic drivers of diseases in people with rare and extreme phenotypes. A phenotype refers to a person’s characteristics or traits that result from the interaction of their genes and their environment.

    This work will help us to identify pathways that are important in certain diseases – or on the flip-side that help people to remain healthy. Ultimately, this approach may present new drug targets to treat diseases in areas where we have leading capabilities: immunology, neurology and bone.

    Among the initiatives currently under way in this area are collaborative projects with Baylor, Duke and Columbia  Universities in the US and Oxford and Cambridge Universities in the UK. This research is already beginning to deliver new genes and disease mechanisms with potentially exciting opportunities to translate scientific knowledge into results for patients.

    For example, the team in Cambridge has identified an individual with relapsing nodular panniculitis, a rare auto-inflammatory phenotype. Working with Professor Eamonn Maher and Dr David Komander of Cambridge University, UCB has discovered a novel gene called OTULIN that causes this potentially fatal disease.

    In a paper published in Cell, a medical research journal, the team has now shown that the OTULIN gene plays an important role in a number of inflammatory and auto-immune pathways. This new knowledge could pave the way for treatments for a number of auto-inflammatory diseases.

    This ground-breaking work is part of UCB’s Genetics Strategy which aims to turn science into patient value. We will continue to push the boundaries of medical science to improve the lives of people with severe diseases.

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