CSTF2 inhibitor

CSTF2 inhibitors represent a targeted therapeutic strategy based on the principle of synthetic lethality. The target, Cleavage stimulation factor subunit 2 (CSTF2), is a protein involved in mRNA 3' end processing. In normal physiology, CSTF2 and its paralogue CSTF2T (Cleavage stimulation factor subunit 2 Tau) are functionally redundant. However, genomic analysis has revealed that certain cancers, including lung adenocarcinoma and melanoma, frequently harbor homozygous deletions of CSTF2T. In these CSTF2T-deficient cells, the function of CSTF2 becomes essential for survival. Research conducted by Boehringer Ingelheim has demonstrated that inhibiting CSTF2 in these specific genetic backgrounds leads to impaired tumor cell growth, providing a potential therapeutic window for treating tumors with CSTF2T loss while sparing healthy tissues.