Biotech company Gain Therapeutics, Inc. announced positive results from an initial study of two Structurally Targeted Allosteric Regulators (STARs), GT-02287 and GT-02329, for the potential treatment of Parkinson’s and Gaucher disease. Using their Site-Directed Enzyme Enhancement Therapy (SEE-Tx™) platform, Gain analyzes the 3D structure of proteins to identify new treatment targets in neurodegenerative disorders.
The target of this study was GCase, a protein that’s encoded by the GBA1 gene. Mutations in the GBA1 gene, which cause the misfolding of GCase, are a well-known genetic risk factor for the development of Parkinson’s disease and other synucleinopathies. Mutations in GBA1 are also a risk factor for Gaucher disease, a rare autosomal storage disorder that puts patients at increased risk for Parkinson’s disease and dementia with Lewy bodies.
Researchers demonstrated that GT-02287 and GT-02329 guided misfolded GCase into its proper shape and restored its activity; in dopaminergic neurons, the compounds reduced levels of phosphorylated and aggregated alpha-synuclein. Gain plans to submit new drug applications for the compounds later this year.
Full study results will be presented at the Michael J. Fox Foundation Innovating From Drug Discovery to the Clinic: Novel Approaches to PD Therapeutic Development webinar in November.