Core 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]

Core Insights - Gain Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing next-generation allosteric small molecule therapies [3][4] - The company will present at The Citizens Life Sciences Conference on May 8, 2025, with CEO Gene Mack participating in one-on-one meetings [1] - Gain's lead drug candidate, GT-02287, is currently in a Phase 1b clinical trial for treating Parkinson's disease and has potential applications in other neurodegenerative diseases [3][4] Company Overview - Gain Therapeutics specializes in discovering and developing allosteric therapies, with a unique approach to creating small molecule modulators that can alter protein function [3][4] - The company utilizes its advanced Magellan™ platform to accelerate drug discovery for challenging disorders, including neurodegenerative diseases and rare genetic disorders [4] Clinical Development - GT-02287 is being evaluated for Parkinson's disease treatment, with further potential in conditions like Gaucher's disease, dementia with Lewy bodies, and Alzheimer's disease [3] - Gain has multiple undisclosed preclinical assets targeting lysosomal storage disorders, metabolic diseases, and solid tumors [3]

Core Insights - Gain Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing next-generation allosteric small molecule therapies [3][4] - The company will present at The Citizens Life Sciences Conference on May 8, 2025, with one-on-one meetings available for interested parties [1][2] Company Overview - Gain's lead drug candidate, GT-02287, is currently in a Phase 1b clinical trial for treating Parkinson's disease, with potential applications in Gaucher's disease, dementia with Lewy bodies, and Alzheimer's disease [3] - The company has multiple undisclosed preclinical assets targeting lysosomal storage disorders, metabolic diseases, and solid tumors [3] Technology and Approach - Gain employs a unique approach to discover novel allosteric small molecule modulators that can restore or disrupt protein function [4] - The Magellan™ platform is utilized to accelerate drug discovery and develop disease-modifying treatments for challenging disorders, including neurodegenerative diseases and rare genetic disorders [4]