GSA-1103

GSA-1103 is an experimental small molecule G-quadruplex interacting drug (GQID) designed to target and stabilize G-quadruplex DNA structures within the promoter regions of key oncogenic drivers, specifically c-Myc and Bcl-2. By interacting with these unique secondary DNA structures, GSA-1103 inhibits the transcription of these genes, which are often overexpressed and considered "undruggable" in various cancers. Research conducted by institutions including the University of Arizona, Arizona State University, and Phoenix Children's Hospital has demonstrated its potential in treating acute myeloid leukemia (AML), where it shows potent growth inhibition in both pediatric and adult AML cell lines with IC50 values ranging from 40 nM to 2 μM.