GT-02287
Pharmaceutical compound
From Wikipedia, the free encyclopedia
GT-02287 is a β-glucocerebrosidase (GCase) activator, positive allosteric modulator, and/or chaperone which is under development for the treatment of Parkinson's disease, Gaucher's disease, solid tumors, and dementia.[1][2][3][4][5][6] It is taken orally.[1]
| Clinical data | |
|---|---|
| Other names | GT02287 |
| Routes of administration | Oral[1] |
| Drug class | β-Glucocerebrosidase (GCase) activator or positive allosteric modulator and/or chaperone |
The drug is under development by Gain Therapeutics.[1][2][3] As of January 2026, it is in phase 1 clinical trials for Parkinson's disease, the preclinical research stage of development for Gaucher's disease and solid tumors, and the research stage of development for dementia.[1][2][3] GT-02287 was also under development for alpha 1-antitrypsin deficiency, globoid cell leukodystrophy, and GM1 gangliosidosis, but development for these indications was discontinued.[1]
The chemical structure of GT-02287 does not yet appear to have been disclosed.[1] However, Gain Therapeutics has patented GCase allosteric chaperones, notably including rexaceract (INN), which is described as a β-glucocerebrosidase positive allosteric modulator and antiparkinsonian agent.[7][8][9] Gain Therapeutics is or was also developing another GCase activator, GT-02329, to treat Gaucher's disease and Parkinson's disease, but no recent development has been reported for this candidate.[10]
See also
- List of investigational Parkinson's disease drugs
- Pariceract (BIA 28-6156; LTI-291)
- Ambroxol