scAAV9-GALT

scAAV9-GALT is a self-complementary adeno-associated virus serotype 9 (scAAV9) gene therapy candidate designed for the treatment of Classic Galactosemia (Type 1 galactosemia). Classic Galactosemia is an autosomal recessive metabolic disorder caused by mutations in the *GALT* gene, which encodes the enzyme galactose-1-phosphate uridylyltransferase. This deficiency leads to the accumulation of toxic metabolites, including galactose-1-phosphate (Gal-1P) and galactitol, resulting in hepatocellular damage, intellectual disability, and motor dysfunction. scAAV9-GALT delivers a functional copy of the *GALT* gene to restore enzyme activity and normalize galactose metabolism. Preclinical studies in mouse models have demonstrated that intravenous administration of scAAV9-GALT can restore GALT activity, reduce toxic metabolite accumulation in both the central nervous system and peripheral tissues, and improve behavioral outcomes.