HaloTag

Gain Therapeutics Announces Preclinical Data Showing Restoration of Enzymatic Function with Novel Allosteric Regulators in GM1 Gangliosidosis Model

Retrieved on: 
Friday, December 1, 2023
Quality of life, Protein, GLB1, GM1, Disorder, PLOS, Brain, Muscle weakness, Doctor of Philosophy, Research, Therapy, Glycogen storage disease, Life expectancy, DSM-IV codes, Paratriathlon, Dystonia, Protein folding, Patient, HaloTag, Pharmaceutical industry

BETHESDA, Md., Dec. 01, 2023 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc., (Nasdaq: GANX), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today 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 β-Galactosidase correctors,” was published in PLOS ONE.

Key Points: 
  • The study, “Validation of a highly sensitive HaloTag-based assay to evaluate the potency of a novel class of allosteric β-Galactosidase correctors,” was published in PLOS ONE.
  • GM1-gangliosidosis is an inherited, progressive disorder characterized by the degeneration of brain and spinal cord cells, leading to muscle weakness, skeletal abnormalities, dystonia, and vision problems.
  • Mutations in the GLB1 gene significantly reduce the activity and function of the lysosomal hydrolase enzyme β-galactosidase (β-Gal) due to protein misfolding.
  • Additionally, there are no available disease-modifying treatments for GM1 gangliosidosis, with available therapies only focused on reducing symptoms and improving quality of life.