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.