Gain Therapeutics(US:GANX) Globenewswire·2025-10-30 11:00Core Insights - Gain Therapeutics, Inc. is presenting its lead drug candidate GT-02287 at the Society for Neuroscience annual meeting, focusing on its potential in treating Parkinson's disease [1][2] Company Overview - Gain Therapeutics is a clinical-stage biotechnology company specializing in the discovery and development of allosteric small molecule therapies [7] - The company is advancing GT-02287, which targets the lysosomal enzyme glucocerebrosidase (GCase) and is currently in a Phase 1b clinical trial for Parkinson's disease [7][5] Drug Candidate Details - GT-02287 is an orally administered small molecule that acts as an allosteric modulator, restoring GCase function impaired by GBA1 mutations or age-related stress [2] - Preclinical studies indicate that GT-02287 can reduce neuroinflammation, neuronal death, and restore motor function in models of both GBA1-PD and idiopathic PD [3][2] Clinical Trial Progress - A Phase 1 study showed favorable safety and tolerability, with increased GCase activity in participants receiving GT-02287 [4] - The ongoing Phase 1b trial aims to evaluate safety and tolerability over 3 months, with early observations suggesting disease-modifying effects [5] Funding and Support - Gain Therapeutics has received funding from The Michael J. Fox Foundation, The Silverstein Foundation, and the Eurostars-2 program, indicating strong support for its Parkinson's disease program [6] Research and Development Approach - The company utilizes its Magellan™ platform to discover novel allosteric small molecule modulators, targeting various disorders including neurodegenerative diseases and rare genetic disorders [8]