ro5-3335

Ro5-3335 is a small molecule benzodiazepine derivative and experimental drug that acts as a selective inhibitor of the RUNX1–CBFβ (core binding factor beta) interaction, thereby repressing RUNX1/CBFB-dependent transcriptional activation. It disrupts the function of RUNX1 and CBFβ, which are crucial transcription factors in hematopoiesis and leukemogenesis. Ro5-3335 shows antiproliferative effects in human CBF leukemia cell lines, selectively kills leukemia cells with CBF fusion proteins, and is effective in animal models for reducing leukemia burden. The drug has also been shown to have potential therapeutic effects in other diseases involving abnormal RUNX1 function, such as cardiac remodeling after myocardial infarction, pulmonary hypertension, retinal angiogenesis disorders, acute kidney injury, and HIV-1 infection (as a Tat inhibitor)[1][2][4][7][10][12][13].

Other names

CBFβ-Runx1 inhibitor II7-chloro-1,3-dihydro-5-(1H-pyrrol-2-yl)-2H-1,4-benzodiazepin-2-onenordazepam derivative