GalK inhibitor (Scenic Biotech)

Scenic Biotech is developing an investigational, first-in-class small-molecule inhibitor targeting galactokinase (GALK) for the treatment of classic galactosemia. This compound is a novel chemical entity (NCE) characterized as an allosteric covalent inhibitor. The therapeutic strategy utilizes a substrate reduction therapy (SRT) approach; by inhibiting GALK—the first enzyme in the Leloir pathway—the drug prevents the conversion of galactose into galactose-1-phosphate (Gal-1-P). The accumulation of Gal-1-P is considered the primary pathogenic driver of the severe clinical manifestations seen in classic galactosemia. By blocking this step, the treatment aims to convert the severe classic phenotype into a condition resembling Type II galactosemia (GALK deficiency), which typically presents with a significantly milder clinical course. The program is currently in the preclinical stage.

Other names

GALK inhibitorGalactokinase inhibitorGALK1 inhibitorGALK-1 inhibitorGALK 1 inhibitorGalactokinase (GalK) inhibitor