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Gain Therapeutics, Inc. Announces Breakthrough Pre-Clinical Data of Novel Small Molecules for the Treatment of Parkinson’s Disease and Gaucher Disease
Study results demonstrate first small molecule approach to significantly reduce phosphorylated and aggregated -synuclein and increase GCase protein levels
About this update from Gain Therapeutics, Inc.
[{"type":"text","content":"Study results demonstrate first small molecule approach to significantly reduce phosphorylated and aggregated -synuclein and increase GCase protein levels with transport to the lysosomes in iPSC dopaminergic neurons Results presented at Michael J. Fox Foundation’s Therapeutic Development Webinar BETHESDA, Md, Nov. 10, 2021 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a biotechnology company directly addressing the urgent need for effective neurodegenerative therapies, today announced data from the Company’s study conducted at the University of Maryland School of Medicine (UMSOM). The study, evaluating two lead Structurally Targeted Allosteric Regulators (STARs) compounds – GT-02287 and GT-02329 – for the treatment of Gaucher and GBA1 Parkinson’s Disease, demonstrated highly statistically significant effects on all tested phenotypes representing a key breakthrough in the potential to treat neurodegenerative disorders characterized by misfolded proteins including Parkinson’s Disease, Alzheimer’s, Gaucher’s Disease and Lewy Body Dementia. Study results were presented at The Michael J. Fox Foundation for Parkinson’s Research’s Innovating from Drug Discover to the Clinic: Novel Approaches to PD Therapeutic Development webinar. Ricardo Feldman, Ph.D., Associate Professor of Microbiology and Immunology at the University of Maryland School of Medicine, stated, \"Our laboratory is using human induced pluripotent stem cells (iPSC) derived from patients with Gaucher and GBA-associated Parkinson’s Disease to test the efficacy of the two lead STAR chaperones developed by Gain Therapeutics. Our studies in iPSC-derived cortical and dopaminergic neurons from neuronopathic Gaucher Disease patients show that these compounds significantly increase the levels of GCase protein, its transport to the lysosome, and its enzymatic activity. In dopaminergic neurons, the two lead STAR chaperones also decrease the levels of the hallmark of Parkinson’s Disease - α-synuclein-p129, demonstrating their potential to treat GBA1-associated Parkinson’s Disease.” “This breakthrough in the ability to restore enzyme function for Parkinson’s Disease and Gaucher’s Disease is unprecedented for a small molecule and will contribute to a major shift in how we think about treating various neurodegenerative and lysosomal storage disea...