Gain Therapeutics, Inc. announced the publication of preclinical data identifying a novel class of small molecule allosteric regulators that demonstrate therapeutic potential for galactosidase beta 1 (GLB1)-related lysosomal storage disorders (LSDs), including GM1 gangliosidosis. The study, "Validation of a highly sensitive HaloTag-based assay to evaluate the potency of a novel class of allosteric b-Galactosidase correctors," was published in PLOS ONE. In the PLOS ONE study, scientists from Gain used Gain's proprietary Site-directed Enzyme Enhancement Therapy (SEE-Tx) technology followed by molecular interaction and ligand binding studies to identify and characterize two structurally targeted allosteric regulators of b-Gal.

A biochemical HaloTag cleavage assay was developed by the researchers from the Institute for Research in Biomedicine and utilized to measure lysosomal delivery capabilities of b-Gal. Results showed that the two compounds (GT-00513 and GT-00413) stabilized, enhanced and restored the key biological lysosomal transport functions of b-Gal and reduced the levels of the intracellular toxic substrate, GM1 gangliosidOSide, in GM1 gangliosidosidosis patient-derived cells.