Gain Therapeutics Presents Preclinical GT-02287 Data at Neuroscience 2025

Core Insights - Gain Therapeutics, Inc. announced promising preclinical data for its lead drug candidate GT-02287, which shows potential as a disease-modifying therapy for Parkinson's disease (PD) by enhancing mitochondrial and lysosomal health [1][2][7] Preclinical Data - GT-02287 demonstrated a reduction in mitochondrial stress and improved neuronal survival in rat dopaminergic neurons treated with MPP+, a mitochondrial toxin [3] - In a mouse PD model, GT-02287 decreased staining for MIRO1, indicating improved mitochondrial health [4] - The drug facilitated the trafficking of glucocerebrosidase (GCase) to mitochondria, enhancing mitochondrial function in patient-derived fibroblasts with GBA1 mutations [5][6] Mechanism of Action - The drug acts throughout the disease cascade resulting from dysfunctional GCase, contributing to neuroprotection by restoring GCase function and reducing neuroinflammation and neuronal death [2][6][7] - GT-02287's effects on mitochondrial health were highlighted as a significant aspect of its mechanism, addressing previously open questions in PD research [2][4] Clinical Development - GT-02287 is currently in a Phase 1b clinical trial for PD, with early observations suggesting disease-modifying effects consistent with preclinical findings [9] - The trial aims to evaluate safety and tolerability after 3 months of dosing, with an extension allowing treatment for up to 12 months [9] Funding and Support - The development of GT-02287 has received funding from notable organizations, including The Michael J. Fox Foundation and the Eurostars-2 joint program [10]