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ABX-10241 is an investigational small molecule developed by Abivax, belonging to a novel class of quinoline derivatives. It is designed to treat chronic inflammatory conditions by modulating RNA splicing. The drug's primary mechanism involves binding to the Cap Binding Complex (CBC) at the 5' end of nascent RNA transcripts. This interaction specifically enhances the splicing and subsequent expression of microRNA-124 (miR-124), a critical regulator of the inflammatory response. Increased levels of miR-124 lead to the systemic downregulation of multiple pro-inflammatory cytokines, including TNF-α, IL-6, and MCP-1, thereby promoting the resolution of inflammation. ABX-10241 is being explored as a potential therapy for inflammatory bowel diseases, such as ulcerative colitis and Crohn's disease, offering a mechanism that aims to restore immune homeostasis by resetting the immune response rather than merely suppressing it.
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