GM1 Gangliosidosis is a hereditary, progressive disease mostly impacting neurons in the brain and spinal cord, caused by mutations in GLB1, the gene that encodes the beta-galactosidase (GLB) enzyme. These mutations result in the misfolding and subsequent dysfunction of GLB, which leads to the toxic substrate accumulation of GM1 ganglioside in organs and tissues. Symptoms include hypotonia, enlargement of spleen and liver, seizures and visual impairment. Gain has developed novel small molecule allosteric regulators of GLB that have been shown to significantly restore GLB function and reduce intracellular toxic substrates in a preclinical model of GM1 gangliosidosis.