Article · Wikipedia archive · Last revised Jul 10, 2026

GT-02287

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. It is taken orally.

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Jul 10, 2026
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GT-02287
Clinical data
Other namesGT02287
Routes of
administration		Oral1
Drug classβ-Glucocerebrosidase (GCase) activator or positive allosteric modulator and/or chaperone

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.123456 It is taken orally.1

The drug is under development by Gain Therapeutics.123 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.123 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

Rexaceract (INNTooltip International Nonproprietary Name) structure. source ↗

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 (INNTooltip International Nonproprietary Name), which is described as a β-glucocerebrosidase positive allosteric modulator and antiparkinsonian agent.789 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

See also

References

References

  1. "GT 02287". AdisInsight. Springer Nature Switzerland AG. 29 January 2026. Retrieved 19 February 2026.
  2. "Delving into the Latest Updates on GT-02287 with Synapse". Synapse. 20 December 2025. Retrieved 19 February 2026.
  3. "GT-02287 Drug Profile". Ozmosi. 1 January 1900. Retrieved 19 February 2026.
  4. Fregno I, Pérez-Carmona N, Rudinskiy M, Soldà T, Bergmann TJ, Ruano A, et al. (May 2025). "Allosteric Modulation of GCase Enhances Lysosomal Activity and Reduces ER Stress in GCase-Related Disorders". International Journal of Molecular Sciences. 26 (9): 4392. doi:10.3390/ijms26094392. PMC 12072338. PMID 40362629.
  5. Pozzi R, De Sciscio M, Bosetti M, Cano-Crespo S, Taylor J, Ignoni T, et al. (November 2025). "Glucocerebrosidase Target Engagement and Therapeutic Plasma and Cerebrospinal Fluid Levels After GT-02287 Administration in Healthy Volunteers". Movement Disorders mds.70111. doi:10.1002/mds.70111. PMID 41194551.
  6. Turner D (8 October 2025). "Early data suggest GT-02287 slows Parkinson's disease". Drug Discovery World (DDW). Retrieved 19 February 2026.
  7. "Heteroaryl compounds and therapeutic uses thereof in conditions associated with the alteration of the activity of beta-glucocerebrosidase". Google Patents. 25 November 2020. Retrieved 19 February 2026.
  8. "4-amino-N-(2,3-dihydro-1H-inden-2-yl)-6-(2-fluoroanilino)pyridine-2-carboxamide". PubChem. Retrieved 19 February 2026.
  9. "Proposed INN: List 134 International Nonproprietary Names for Pharmaceutical Substances (INN)" (PDF). WHO Drug Information. 39 (4): 1270–1271. 2025.
  10. "GT 02329". AdisInsight. Springer Nature Switzerland AG. 28 October 2025. Retrieved 19 February 2026.
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