GSK4172239

GSK4172239 is a potent, selective, small molecule inhibitor of DNA methyltransferase 1 (DNMT1) currently in Phase 1 clinical development for the treatment of sickle cell disease (SCD). Developed through a collaboration between GSK and Cancer Research Horizons (formerly Cancer Research UK's commercial arm), the compound works by inhibiting the epigenetic silencing of the γ-globin gene. This inhibition leads to the induction of fetal hemoglobin (HbF) production, which prevents the polymerization of sickle hemoglobin (HbS) and reduces the clinical complications of SCD. Unlike non-selective hypomethylating agents like decitabine, GSK4172239 is designed for oral administration and improved selectivity for DNMT1, potentially offering a more targeted and convenient therapeutic option for patients with hemoglobinopathies.

Other names

GSK4172239GSK-4172239GSK 4172239