Gain Therapeutics Email Format

Biotechnology ResearchMaryland, United States11-50 Employees

Gain Therapeutics is a clinical-stage biotechnology company focused on discovering and developing allosteric small-molecule therapies. Its lead candidate, GT-02287, is being evaluated for Parkinson’s disease with or without a GBA1 mutation, with Phase 1 data in healthy volunteers indicating favorable safety and tolerability, expected systemic exposure within the therapeutic range, central nervous system exposure, and evidence of target engagement and modulation of the GCase enzyme. The company aims to restore or disrupt protein function through allosteric modulation, supported by its Magellan platform to accelerate drug discovery targeting neurodegenerative diseases, rare genetic disorders, and oncology.
Based in Bethesda, Maryland, the organization operates with a small to mid-sized team focused on translating basic biology into potential therapies. In March 2026, Gain announced preclinical updates, including a new chemical series of allosteric GCase modulators led by GT-04686 that are ready for IND-enabling studies.

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