Gain Therapeutics(US:GANX) Newsfilter·2024-05-30 11:00Core Insights - Gain Therapeutics, Inc. announced that a late-breaking abstract on GT-02287 has been accepted for presentation at the FENS Forum 2024, highlighting cognitive performance improvements in a preclinical model of GBA1 Parkinson's disease [1][2] Company Overview - Gain Therapeutics is a clinical-stage biotechnology company focused on developing next-generation allosteric small molecule therapies [6] - The lead drug candidate, GT-02287, is in clinical development for GBA1 Parkinson's disease and is currently undergoing a Phase 1 clinical trial [6] Drug Candidate Details - GT-02287 is an orally administered, brain-penetrant small molecule that acts as an allosteric protein modulator, restoring the function of the lysosomal enzyme glucocerebrosidase (GCase) impaired by GBA1 gene mutations [4] - In preclinical models, GT-02287 has shown to restore GCase function, reduce aggregated α-synuclein, decrease neuroinflammation and neuronal death, increase dopamine levels, and improve both motor function and cognitive performance [4] - The drug also significantly reduced plasma neurofilament light chain (NfL) levels, a biomarker for neurodegeneration [4] Funding and Support - Gain Therapeutics has received funding support from The Michael J. Fox Foundation for Parkinson's Research, The Silverstein Foundation for Parkinson's with GBA, and the Eurostars-2 joint program co-funded by the European Union Horizon 2020 research and Innosuisse [5] Research and Development Platform - The company utilizes the Magellan™ drug discovery platform, which leverages AI-supported structural biology and supercomputer-powered models to identify novel allosteric binding sites on disease-implicated proteins [7] - This platform enhances the original SEE-Tx® platform with new AI and machine-learning tools, allowing access to a vast chemical space of over five trillion compounds [7][8] Event Details - The poster presentation at the FENS Forum will take place on June 27, 2024, at 11:30 am, focusing on the improvements in daily living activities and cognitive performance due to GT-02287 [2]