Gain Therapeutics(US:GANX) Globenewswire·2025-05-12 11:30Core Insights - Gain Therapeutics, Inc. announced new evidence supporting GT-02287's neuroprotective effects and potential as a disease-modifying therapy for Parkinson's disease at the IAPRD 30th World Congress [1][3] Company Overview - Gain Therapeutics is a clinical-stage biotechnology company focused on developing allosteric small molecule therapies, with GT-02287 as its lead candidate for treating Parkinson's disease [11][12] - GT-02287 is designed to restore the function of the lysosomal enzyme glucocerebrosidase (GCase), which is often impaired due to mutations in the GBA1 gene, a common genetic abnormality associated with Parkinson's disease [5][11] Research and Development - Recent preclinical data indicate that GT-02287 improves lysosomal and mitochondrial function, reduces α-synuclein aggregation, and promotes neuronal survival by preventing apoptosis signals [3][5] - The drug has shown a disease-modifying effect in preclinical models of both GBA1-PD and idiopathic PD, suggesting it may slow or stop disease progression [6][11] - A Phase 1 study demonstrated favorable safety and tolerability, with over a 50% increase in GCase activity in participants receiving GT-02287 [7][8] Clinical Trials - GT-02287 is currently being evaluated in a Phase 1b clinical trial for Parkinson's disease, focusing on safety and tolerability after three months of dosing [8] - The trial is enrolling participants across seven sites in Australia [8] Funding and Support - Gain Therapeutics has received funding support from The Michael J. Fox Foundation for Parkinson's Research and other organizations to advance its lead program [9][10]