GT-00513

GT-00513 is a preclinical small molecule allosteric regulator of lysosomal beta-galactosidase (β-Gal), discovered by Gain Therapeutics using its Site-directed Enzyme Enhancement Therapy (SEE-Tx) platform as part of a program targeting GLB1-related lysosomal storage disorders such as GM1 gangliosidosis.[1][10][11][12] By binding to β-Gal at a structurally targeted allosteric site outside the catalytic pocket, GT-00513 acts as a pharmacological chaperone that stabilizes misfolded mutant enzyme, enhances its lysosomal trafficking, restores enzymatic activity, and reduces intracellular accumulation of GM1 ganglioside in patient-derived and other disease model cells.[1][10][11] These data support further development of GT-00513 or related compounds as potential disease-modifying therapies for GM1 gangliosidosis and other GLB1-deficiency disorders.[1][3][10][11][12]