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Gain Therapeutics, Inc. Announces Positive Topline Data in Patient-derived iPSC Study Evaluating STAR Compounds as a Treatment for Gaucher and Parkinson’s Disease
GT-02287and GT-02329 compounds display positive effects on GCase activityand related parameters---Study results demonstrate an increase in GCase protein
About this update from Gain Therapeutics, Inc.
[{"type":"text","content":"GT-02287and GT-02329 compounds display positive effects on GCase activityand related parameters---Study results demonstrate an increase in GCase protein levels with transport to the lysosomes and decrease of glucosylceramide and α-synuclein-p129 levels---Comprehensive study results to be presented at Michael J. Fox Foundation’s upcoming Innovating from Drug Discovery to the Clinic: Novel Approaches to PD Therapeutic Development webinar---IND-initiating study planned for Q4’21 BETHESDA, Md., Sept. 08, 2021 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a biotechnology company focused on identifying and optimizing allosteric binding sites never before targeted in neurodegenerative diseases and lysosomal storage disorders, today announced topline 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 positive effects on all tested phenotypes. 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 GD 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 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 α-synuclein-p129, demonstrating their potential to treat GBA1-associated Parkinson’s Disease.” “These data are extremely exciting, as it further demonstrates the potential of GT-02287 and GT-02329 and expands the body of evidence supporting our Site-Directed Enzyme Enhancement Therapy (SEE-Tx™) drug discovery platform,” said Eric Richman, Chief Executive Officer of Gain. “We plan to fully evaluate the results of this study and present a complete data set on the upcoming Michael J. Fox Foundation’s Innovating from Drug Discovery to the Clinic: Novel Approaches to PD The...