ASP210

ASP210 is a potent oligonucleotide-based inhibitor designed to target BCR-ABL1 mRNA. It induces cell death selectively in BCR-ABL1-positive cells, including those resistant to tyrosine kinase inhibitors (TKIs), such as imatinib and dasatinib, regardless of T315I mutation status. ASP210 works by suppressing the expression of the leukemia driver protein at the mRNA level, leading to apoptosis in chronic myeloid leukemia (CML) cells that are dependent on BCR-ABL1 signaling. Importantly, it shows minimal effect on BCR-ABL1-negative healthy cells, indicating high selectivity and a favorable safety profile. The drug has demonstrated dose-dependent cytoreductive effects in vitro and is considered a promising therapeutic avenue for patients with CML who fail to respond to TKI therapy[1][2][6].