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GT-02287

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GT-02287
Clinical data
Other namesGT02287
Routes of
administration		Oral[1]
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.[1][2][3][4][5][6] It is taken orally.[1]

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]

Rexaceract (INNTooltip International Nonproprietary Name) structure.

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.[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

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References

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  1. ^ a b c d e f g "GT 02287". AdisInsight. Springer Nature Switzerland AG. 29 January 2026. Retrieved 19 February 2026.
  2. ^ a b c "Delving into the Latest Updates on GT-02287 with Synapse". Synapse. 20 December 2025. Retrieved 19 February 2026.
  3. ^ a b c "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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