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guadecitabine

Development stage
Phase 3
Lead developer
Astex Pharmaceuticals
Modality
Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules, Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules, DNA Intercalators/Alkylators → Nucleic Acid-Directed Small Molecules → Small Molecules
Administration
Subcutaneous, Intravenous
01

Overview

Guadecitabine is a next-generation small molecule hypomethylating agent and dinucleotide antimetabolite composed of decitabine linked via a phosphodiester bond to deoxyguanosine. It acts as an inhibitor of DNA cytosine 5-methyltransferase 1 (DNMT1), leading to the reversal of aberrant DNA methylation and reactivation of silenced tumor suppressor genes. Guadecitabine was developed primarily for hematological malignancies such as acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS), with additional investigation in various solid tumors. It was designed for subcutaneous administration to provide prolonged exposure compared to decitabine[2][5][6][7].

Other names
decitabine deoxyguanosinedecitabine deoxyguanosine dinucleotideguadecitabine sodium
02

Targets

DNMT1 (DNA (cytosine-5)-methyltransferase 1)

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