Gain Therapeutics, Inc.
June 16, 2025
Company Presentation
Other
Gain Therapeutics, Inc. is a clinical-stage biotechnology company leading the discovery and development of next generation allosteric therapies. Gain’s lead drug candidate, GT-02287 is currently being evaluated for the treatment of Parkinson’s disease with or without a GBA1 mutation in a Phase 1b clinical trial. GT-02287 has further potential in Gaucher’s disease, dementia with Lewy bodies, and Alzheimer’s disease. Gain has multiple undisclosed preclinical assets targeting lysosomal storage disorders, metabolic diseases, and solid tumors.
Gain’s unique approach enables the discovery of novel, allosteric small molecule modulators that can restore or disrupt protein function. Deploying its highly advanced Magellan™ platform, Gain is accelerating drug discovery and unlocking novel disease-modifying treatments for untreatable or difficult-to-treat disorders including neurodegenerative diseases, rare genetic disorders, and oncology.
Company HQ City:
Bethesda
Company HQ State:
Maryland
Company HQ Country:
United States
Year Founded:
2017
Lead Product in Development:
GT-02287, is in clinical development for the treatment of Parkinson’s disease (PD) with or without a GBA1 mutation. The orally administered, brain-penetrant small molecule is an allosteric enzyme modulator that restores the function of the lysosomal enzyme glucocerebrosidase (GCase) which becomes misfolded and impaired due to mutations in the GBA1 gene, the most common genetic abnormality associated with PD, or other age-related stress factors.
CEO
Gene Mack, President and CEO
Development Phase of Lead Product
Phase I
Exchange
NASDAQ-GM
Ticker
NASDAQ: GANX
When you expect your next catalyst update?
First analysis of biomarker data from the Phase 1b study is expected by the end of the second quarter of 2025. As part of the first analysis , various biomarkers from blood of participants with either idiopathic PD or GBA1 will be evaluated.
What is your next catalyst (value inflection) update?
June 2025
Primary Speaker
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