Core Viewpoint - Gain Therapeutics, Inc. is advancing its lead drug candidate GT-02287 for the treatment of Parkinson's disease, with a focus on its disease-modifying potential and biomarker data from a Phase 1b clinical study [1][2]. Company Overview - Gain Therapeutics, Inc. is a clinical-stage biotechnology company specializing in the discovery and development of next-generation allosteric therapies [10]. - The company is currently evaluating GT-02287 in a Phase 1b clinical trial for Parkinson's disease, targeting patients with or without a GBA1 mutation [10]. Drug Candidate Details - GT-02287 is an orally administered, brain-penetrant small molecule that acts as an allosteric enzyme modulator, restoring the function of the lysosomal enzyme glucocerebrosidase (GCase) [5]. - Preclinical studies have shown that GT-02287 can restore GCase enzymatic function and reduce various pathologies associated with Parkinson's disease, including neuroinflammation and neuronal death [5][6]. - The drug has demonstrated a disease-modifying effect in preclinical models, suggesting potential to slow or stop the progression of Parkinson's disease [6]. Clinical Study Insights - The Phase 1 study of GT-02287 in healthy volunteers indicated favorable safety and tolerability, with target engagement reflected in increased GCase activity [7]. - The ongoing Phase 1b clinical trial has enrolled participants across seven sites in Australia, focusing on the safety and tolerability of GT-02287 after three months of dosing [8]. - An extension of the Phase 1b study allows participants to continue treatment for up to 12 months [8]. Funding and Support - Gain Therapeutics has received funding support for its lead program from The Michael J. Fox Foundation for Parkinson's Research and The Silverstein Foundation for Parkinson's with GBA, among others [9].