GT-02216 

GT-02216 can bind to GCase allosterically and enhance its activity. GT-02216 enhances the activity of GCase in primary human fibroblasts dose-dependently reduces the accumulation of its substrate, hexosylsphingosine (HexCer). GT-02216 reduces the Tau accumulation in mutant GBA1 fibroblasts. GT-02216 can be used for the study of Parkinson’s disease^[1].

GT-02216 binds to GCase protein in a dose-dependent manner at both acidic pH 5.0 (K_D = 54.2 μM) and neutral pH 7.4 (K_D = 55 μM) with similar binding affinity^[1].
GT-02216 (12.5 μM, 4 days) shows significant enhancement of GCase activity in mutant GBA1 fibroblasts as well as in GBA1^WT(XY) fibroblasts, except for the GBA1^{L444P/L444P(I)β} cell line, which shows undetectable GCase activity^[1].
GT-02216 (1.56-25 μM, 10 days) decreases the accumulation of HexCer in both GBA1^{L444P/L444P(I)α} and GBA1^WT(XX) cells in a dose-dependent manner^[1].
GT-02216 (4 days) rescues GCase activity in a dose-dependent manner in Tau-GBA1^{L444P/L444P(I)α} fibroblasts and elicites a dose-dependent increase in GCase activity also in Tau-GBA1^WT(XX) fibroblasts^[1].
GT-02216 (1.56-25 μM, 4 days) can reduce tau accumulation both at basal (no seeds) or in the presence of tau seeds in a dose-dependent manner in tau-GBA1^{L444P/L444P(I)α} and tau-GBA1^WT(XX)fibroblasts^[1].
GT-02216 (12.5 μM, 4 days) reversed the increase in LAMP1-positive DOs in Tau-GBA1^{L444P/L444P(I)α} and Tau-GBA1^WT(XX) fibroblasts after Tau seed treatment^[1].
GT-02216 (0-3 μM, 48 h) effectively rescues cell viability in the TauO-challenged hippocampal neurons, demonstrating its neuroprotective potential against TauO-induced neurotoxicity^[1].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

389.45

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2648407-76-3

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• [1]. Ciccaldo M, et al. A novel allosteric GCase modulator prevents Tau accumulation in GBA1^WT and GBA1^L444P/L444P cellular models. Sci Rep. 2025 May 21;15(1):17646.  [Content Brief]