Gain Therapeutics (NASDAQ:GANX) is a clinical-stage biotechnology company dedicated to discovering and developing novel small-molecule therapies targeting protein misfolding and aggregation pathways. Utilizing a proprietary structure-based drug discovery engine, the company focuses on identifying allosteric sites that modulate protein function, with the aim of delivering first-in-class treatments for rare neurodegenerative disorders. Its research framework leverages advanced computational modeling and high-throughput screening to accelerate the design of molecule candidates with optimized efficacy and safety profiles.
The company’s core discovery platform integrates artificial intelligence, molecular dynamics simulations and crystallography data to pinpoint cryptic binding pockets on disease-relevant proteins. This approach enables Gain Therapeutics to generate highly selective modulators that correct dysfunctional protein behavior rather than simply inhibiting activity. By exploiting allosteric mechanisms, the pipeline seeks to minimize off-target effects and overcome challenges associated with more conventional drug discovery modalities.
Gain Therapeutics’ lead program is advancing a small-molecule therapeutic for GM2 gangliosidosis, encompassing Tay-Sachs and Sandhoff diseases. Preclinical studies have demonstrated promising pharmacodynamic and pharmacokinetic profiles, paving the way for clinical evaluation. Beyond GM2 gangliosidosis, the company is exploring additional neurometabolic and neurodegenerative targets, including Parkinson’s disease and lysosomal storage disorders. Collaborations with academic institutions and patient advocacy groups support its translational efforts and help inform clinical trial design.
Founded in 2018 and headquartered in Zug, Switzerland, Gain Therapeutics maintains research operations in North America and Europe. The company is led by CEO Andrea Piumatti, whose background spans biotech leadership and drug development, alongside a management team with extensive experience in medicinal chemistry, computational biology and clinical research. Guided by a board of directors comprising seasoned industry executives and scientific advisors, Gain Therapeutics is positioned to advance its innovative platform toward delivering transformative treatments for patients with underserved neurological conditions.
