Gain Therapeutics(US:GANX) Newsfilter·2024-06-27 12:35Core Insights - Gain Therapeutics, Inc. presented a poster at the FENS Forum 2024, highlighting the positive effects of GT-02287 on cognitive performance and daily living activities in a mouse model of GBA1 Parkinson's disease [15][1][3] Group 1: Company Overview - Gain Therapeutics is a clinical-stage biotechnology company focused on discovering and developing next-generation allosteric small molecule therapies [19] - The company's lead drug candidate, GT-02287, is designed to treat GBA1 Parkinson's disease and is currently undergoing a Phase 1 clinical trial [19][3] Group 2: Drug Development and Mechanism - GT-02287 is an orally administered small molecule that acts as an allosteric protein modulator, restoring the function of the glucocerebrosidase (GCase) enzyme, which is impaired due to GBA1 gene mutations [3][19] - In preclinical studies, GT-02287 has shown to restore GCase function, reduce aggregated α-synuclein, decrease neuroinflammation, and improve both motor function and cognitive performance [3][1] Group 3: Research Findings - The study demonstrated that GT-02287 improved nest-building and motor performance in a GBA1-PD mouse model, indicating its potential to enhance complex behaviors related to cognitive function [1][3] - Biomarker changes, including reduced plasma neurofilament light chain (NfL) levels, were observed, supporting the drug's efficacy in neurodegeneration [3][1] Group 4: Future Directions - The company aims to evaluate GT-02287 in patients suffering from GBA1 Parkinson's disease, emphasizing the need for effective treatments for this chronic condition [8][15] - Gain Therapeutics is committed to engaging with the neuroscience community to further discuss its innovative approach to treating GBA1 Parkinson's disease [8][15]