Gain Therapeutics to Present GT-02287 Preclinical Data in Parkinson’s Models at Neuroscience 2024

Gain Therapeutics, Inc., a clinical-stage biotech company, recently unveiled key presentations at Neuroscience 2024 that highlighted preclinical data for GT-02287, their leading drug candidate for Parkinson's disease (PD), at the McCormick Place Convention Center in Chicago from October 5-9, 2024.

One of the studies presented by Natalia Perez-Carmona focused on GT-02287's ability to inhibit Tau accumulation in a cellular model. This research was carried out with the assistance of Professor Paolo Paganetti's team at the Laboratories for Translational Research of the Ente Ospedaliero Cantonale in Bellinzona, Switzerland, and backed by an Innosuisse-Swiss Innovation Agency grant involving the Università della Svizzera italiana-affiliated Institute for Research in Biomedicine.

Another pivotal presentation by Joanne Taylor demonstrated that GT-02287 exhibits disease-modifying properties in models of Parkinson’s disease, both those with and without the GBA1 mutation. Perez-Carmona also shared findings on how GT-02287 improves mitochondrial function and offers neuroprotective benefits in GBA1-Parkinson's disease models.

GT-02287 stands out as Gain Therapeutics' leading drug candidate for treating Parkinson's disease, regardless of the presence of a GBA1 mutation. This orally administered, brain-penetrant small molecule is designed to restore glucocerebrosidase (GCase) function. Mutations in the GBA1 gene are a common genetic contributor to Parkinson's disease, causing GCase to become misfolded and dysfunctional. GT-02287 targets this issue, aiming to restore GCase function, reduce α-synuclein aggregation, decrease neuroinflammation and neuronal death, elevate dopamine levels, and improve both motor and cognitive functions. Moreover, the drug has shown potential in reducing plasma neurofilament light chain (NfL) levels, a promising biomarker for neurodegeneration.

The preclinical data underscore GT-02287's promise, particularly in GBA1-PD mouse models. Data presented at the FENS Forum 2024 in June highlighted noteworthy improvements in both cognitive and motor functions following the administration of GT-02287, suggesting its potential to decelerate Parkinson’s disease progression.

Gain Therapeutics has received substantial support for its Parkinson’s disease program from The Michael J. Fox Foundation for Parkinson’s Research and The Silverstein Foundation for Parkinson’s with GBA. Additional backing comes from the Eurostars-2 joint program, co-funded by the European Union Horizon 2020 research initiative and Innosuisse – Swiss Innovation Agency.

Gain Therapeutics, Inc., is a clinical-stage biotech company at the forefront of developing next-generation allosteric therapies. Their flagship drug, GT-02287, is currently in Phase 1 clinical trials for Parkinson’s disease, with or without GBA1 mutations. The company leverages a proprietary AI-supported structural biology platform, Magellan™, which utilizes advanced algorithms and supercomputer-powered models to identify novel allosteric binding sites on disease-related proteins. This innovative platform helps discover small molecule modulators to restore or disrupt protein function, paving the way for new disease-modifying treatments for challenging disorders such as neurodegenerative diseases, rare genetic conditions, and oncology.

Gain Therapeutics employs a highly advanced approach, combining AI and machine learning tools with virtual screening capabilities. This method explores vast chemical spaces containing over five trillion compounds to identify and select appropriate small molecule candidates for experimental validation, thereby accelerating the drug discovery process and addressing previously untreatable or difficult-to-treat diseases.