BP1001 + venetoclax + decitabine

BP1001 is a neutral-charge, liposome-incorporated antisense oligodeoxynucleotide that targets the translation initiation site of the GRB2 transcript, thereby downregulating growth factor receptor-bound protein 2 (Grb2) expression. This inhibition disrupts signaling pathways critical for cancer cell survival and proliferation. BP1001 is developed using DNAbilize technology by Bio-Path Holdings and is being investigated primarily for hematologic malignancies such as acute myeloid leukemia (AML) and chronic myelogenous leukemia (CML). In clinical studies, BP1001 has been combined with venetoclax—a BCL-2 inhibitor—and decitabine—a hypomethylating agent—to enhance anti-leukemic efficacy. The combination has shown encouraging response rates in both newly diagnosed and relapsed/refractory AML patients without additional drug-related toxicity beyond what is expected from standard therapies[2][6]. Venetoclax acts by inhibiting B-cell lymphoma 2 (BCL-2) to promote apoptosis in malignant cells, while decitabine incorporates into DNA to inhibit DNA methyltransferase, leading to hypomethylation and reactivation of tumor suppressor genes.

Other names

liposomal Grb2 antisense oligonucleotide + venetoclax + decitabine