Cap Binding Complex (CBC) (CBC)

The miR-124 biogenesis machinery refers to the cellular components responsible for the production of microRNA-124 (miR-124), a key regulator of the inflammatory response and neuronal development. The primary therapeutic target within this machinery is the nuclear Cap Binding Complex (CBC), a heterodimer consisting of CBP80 (NCBP1) and CBP20 (NCBP2) that binds to the 5' cap of RNA polymerase II transcripts (Abivax, 2024; NIH, 2020). Small molecules like obefazimod (ABX464) bind to the CBC and stabilize its interaction with the protein ARS2 (SRRT), which in turn promotes the selective splicing of the long non-coding RNA LINC00599 (Patsnap, 2026; Vautrin et al., 2019). This enhanced splicing increases the production of mature miR-124, which subsequently downregulates pro-inflammatory cytokines such as TNF-alpha, IL-6, and CCL2 by targeting their mRNAs (Abivax, 2023; Cancer.gov, 2024). This pathway is a major focus for treating chronic inflammatory diseases, particularly ulcerative colitis and Crohn's disease, where miR-124 is often downregulated (Abivax, 2026; NIH, 2023). Additionally, the CBC-mediated modulation of RNA processing has shown potential in inhibiting HIV replication by interfering with the export of unspliced viral RNA (NIH, 2020; Cancer.gov, 2024).

Other names

miR-124 biogenesis machineryCBC-ARS2 complexCBP80/CBP20 complexNuclear cap-binding protein complexNCBP1/NCBP2 complex

Stabilization of the Cap Binding Complex (CBC) and ARS2 interaction to enhance the splicing of LINC00599, resulting in increased miR-124 production.