NCBP1/NCBP2 (nuclear cap-binding complex)

Class

Small-molecule CBC modulator (promotes miR-124 maturation)

Notes

Source: https://www.vbdata.cn/intelDetail/21242 ; https://clinicaltrials.gov/study/NCT07229950 — Chinese secondary-source reporting, not yet cross-checked against a primary Hengrui filing (HRS-7085; researched 2026-08-08)

NCBP1 and NCBP2 together form the nuclear cap-binding complex (CBC), a heterodimer that binds the 5' cap structure added to newly transcribed RNA molecules. This complex is a central hub in RNA metabolism, coordinating splicing, nuclear export, and translation of messenger RNA, but it also participates in the early steps of processing certain small regulatory RNAs called microRNAs. One such microRNA is miR-124, which is normally abundant in neurons and acts as a tumor suppressor by silencing genes that drive cell proliferation, invasion, and stem-like behavior in various tissues. In several cancers, miR-124 levels are reduced, allowing those growth-promoting genes to remain active. A small molecule that enhances CBC's role in miR-124 maturation offers a strategy to restore levels of this protective microRNA without delivering it directly as a synthetic oligonucleotide or gene therapy. This approach is relevant to oncology, where reactivating lost tumor-suppressor pathways is a long-standing goal, and potentially to neurological conditions where miR-124 supports neuronal identity and differentiation. Because CBC also governs broad, essential RNA processing functions across nearly all cell types, the therapeutic challenge lies in nudging its activity toward a specific microRNA pathway rather than causing widespread disruption of normal gene expression.