sodium 2,2-dimethylbutyrate

Sodium 2,2-dimethylbutyrate (HQK-1001) is an orally administered small molecule and a short-chain fatty acid derivative (SCFAD). It has been investigated primarily as an inducer of fetal hemoglobin (HbF) production and erythropoiesis. The drug acts by stimulating γ-globin gene expression in erythroid cells, which increases the production of fetal hemoglobin—a therapeutic strategy for treating β-globin disorders such as sickle cell disease and β-thalassemia. Clinical studies have shown that HQK-1001 can induce HbF at tolerable doses with a favorable safety profile[1][6][8]. The compound has reached phase II clinical trials for these indications.

Other names

sodium 2,2-dimethylbutyratedimebutate sodium2,2-dimethylbutyric acid2,2-dimethylbutanoic acid