Bethesda, United States
Gain Therapeutics, founded in 2017, is a clinical-stage biotechnology company spearheading the development of next-generation allosteric therapies. Its lead drug candidate, GT-02287, is under evaluation for Parkinson’s disease, targeting patients with or without a GBA1 mutation. Phase 1 results in healthy volunteers have shown promising safety profiles, therapeutic-range plasma exposure, and effective CNS engagement.
Deploying its state-of-the-art Magellan™ platform, Gain Therapeutics is pioneering efforts in discovering novel, allosteric small-molecule modulators to influence protein function. This innovation is poised to unlock therapies for previously intractable diseases, including neurodegenerative disorders, rare genetic disorders, and oncology.
The company recently secured a $7.00M Post IPO Equity investment on 01 July 2025, reflecting strong confidence from investors in its strategic approach to transformative drug discovery.
