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Blau Syndrome (Blau) is a granulomatous auto-inflammatory disease caused by mutations in NOD2 that have been proposed to result in phosphorylation of RIP2 kinase and the production of pro-inflammatory cytokines. Such monogenic diseases can bring to light pathways that are also likely to be involved in more genetically complex diseases. For example, increased RIP2 phosphorylation has been observed in inflammatory bowel disease (IBD), although the role of RIP2 in IBD has not been determined. We are developing a first-in-class, highly potent and selective inhibitor of RIP2 kinase, which may provide therapeutic benefit in both Blau and IBD.

OR3-001 – RIP2 kinase is activated in Blau Syndrome and IBD