Gain Therapeutics, Inc.

Gain Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to the discovery and development of next-generation allosteric small molecule therapies. The company's mission is to create innovative medicines that address debilitating neurodegenerative diseases, rare genetic disorders, and cancer. Headquartered in Bethesda, Maryland, USA, Gain Therapeutics also maintains operations in Switzerland and Spain.

At the core of its operations is the proprietary Magellan™ drug discovery platform (formerly SEE-Tx™), which integrates AI-supported structural biology, advanced algorithms, and supercomputer-powered physics-based models. This platform enables the identification of novel allosteric binding sites on disease-implicated proteins, facilitating the discovery of small molecule modulators that can restore or disrupt protein function. The company's lead drug candidate, GT-02287, is an allosteric regulator of GCase currently in development for Parkinson's disease, specifically GBA1-Parkinson's disease, with potential applications extending to Gaucher disease, dementia with Lewy bodies, and Alzheimer's disease. Gain Therapeutics also has a pipeline of preclinical assets targeting various lysosomal storage disorders, metabolic diseases, and solid tumors.

Gene Mack serves as the Chief Executive Officer and President, having been appointed to the role in January 2025. Gain Therapeutics became a publicly traded company on March 18, 2021, with its shares listed on NASDAQ under the ticker symbol GANX. Recent corporate developments include the announcement of positive interim data from its Phase 1b clinical study of GT-02287 for Parkinson's disease, demonstrating central nervous system target engagement and biomarker effects. The company anticipates initiating a placebo-controlled Phase 2 trial in the third quarter of 2026. Gain Therapeutics also reported its financial results for the fourth quarter and full year 2025 in March 2026.