GT-00413

GT-00413 is a preclinical, orally bioavailable small-molecule allosteric modulator of lysosomal beta-galactosidase (β-Gal) being developed by Gain Therapeutics for the treatment of GLB1-related lysosomal storage disorders, particularly GM1 gangliosidosis.[1][3][6][8] Discovered using Gain’s Site-directed Enzyme Enhancement Therapy (SEE-Tx) platform, GT-00413 binds an allosteric site on misfolded β-Gal to stabilize the enzyme, enhance its lysosomal transport, and restore catalytic function, thereby reducing intracellular accumulation of toxic GM1 ganglioside and related substrates in patient-derived and canine fibroblast models.[1][6][7][8] As part of a novel class of structurally targeted allosteric regulators, GT-00413 is intended as a disease-modifying therapy that addresses the underlying enzymatic defect in neurodegenerative and congenital manifestations of GM1 gangliosidosis, with development currently at the preclinical stage.[1][3][4][6][8]