GC2025A

GC2025A is an investigational oral chaperone therapy developed for the treatment of GM1 gangliosidosis, a rare neurodegenerative lysosomal storage disorder caused by autosomal recessive deficiency of the enzyme β-Galactosidase 1 (GLB1). The drug is designed to reduce pathological accumulation of GM1 ganglioside in the brain. In non-clinical studies, oral administration of GC2025A to animal models resulted in a reduction of more than 70% in brain GM1 levels over seven days. There are currently no effective treatments available for GM1 gangliosidosis, which is characterized by early-onset neurological symptoms such as seizures and muscle weakness[1][6].

Brand names

GC2025AGC-2025AGC 2025A

Other names

GC2025AGC-2025AGC 2025A