Gain Therapeutics Announces Oral Poster Presentation at IAPRD 2025 30th World Congress on Parkinson's Disease and Related Disorders

Core Viewpoint - Gain Therapeutics, Inc. is advancing its lead drug candidate GT-02287 for the treatment of Parkinson's disease, showcasing promising preclinical and early clinical data that suggest potential disease-modifying effects [1][3][4]. Company Overview - Gain Therapeutics, Inc. is a clinical-stage biotechnology company focused on discovering and developing next-generation allosteric therapies [7]. - The company is leveraging its Magellan™ platform to accelerate drug discovery for various difficult-to-treat disorders, including neurodegenerative diseases and rare genetic disorders [8][9]. Drug Candidate Details - GT-02287 is an allosteric enzyme modulator aimed at restoring the function of the lysosomal enzyme glucocerebrosidase (GCase), which is often impaired in Parkinson's disease due to GBA1 mutations [2][7]. - The drug is currently in a Phase 1b clinical trial, evaluating its safety and tolerability in patients with Parkinson's disease [5]. Clinical Data - Preclinical studies have shown that GT-02287 can restore GCase activity, reduce neuroinflammation, and protect dopaminergic neurons from mitochondrial toxins [2][3]. - A Phase 1 study in healthy volunteers indicated favorable safety and tolerability, with a greater than 50% increase in GCase activity observed at clinically relevant doses [4]. Funding and Support - Gain Therapeutics has received funding support from notable organizations, including The Michael J. Fox Foundation for Parkinson's Research and the Eurostars-2 joint program, which is co-funded by the European Union [6].