BETHESDA, Md. - Gain Therapeutics, Inc. (NASDAQ: GANX) has announced new data from a preclinical study indicating that its lead drug candidate, GT-02287, showed improvements in cognitive performance in a model of GBA1 Parkinson’s disease. The study's findings are set to be presented at the upcoming FENS Forum 2024, a prominent neuroscience congress in Europe.
The data revealed that GT-02287, a glucocerebrosidase (GCase) enhancer, not only improved cognitive functions but also activities of daily living in a preclinical model of the disease. The GBA1 gene mutation, a common genetic link to Parkinson’s disease, causes misfolding and impairment of the GCase enzyme. GT-02287 is designed to restore this enzyme's function.
In the preclinical trials, the compound also showed a reduction in neurofilament light chain levels, a potential biomarker for neurodegeneration. The improvements in motor function and dopamine levels were additional positive outcomes observed.
Gain Therapeutics' approach involves leveraging AI-supported structural biology and proprietary algorithms to identify novel allosteric binding sites on proteins implicated in diseases. The company's Magellan™ drug discovery platform is an advancement of its SEE-Tx® technology, now enhanced with AI and machine learning tools.
The research has garnered support from The Michael J. Fox Foundation for Parkinson’s Research and The Silverstein Foundation for Parkinson’s with GBA, among others. As GT-02287 progresses through clinical development, its potential therapeutic benefits are being closely monitored, particularly as it is currently undergoing a Phase 1 clinical trial.
The press release also provided a cautionary note regarding forward-looking statements, which are subject to various risks and uncertainties that could cause actual results to differ materially from those projected.
This article is based on a press release statement and aims to report the facts without endorsing any claims. The presentation at FENS Forum 2024 will provide a more detailed insight into GT-02287’s efficacy and its potential impact on treating GBA1 Parkinson’s disease.
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