Gain Therapeutics IncGANX announced the presentation of new preclinical data evaluating one of its lead Structurally Targeted Allosteric Regulator (STAR) compounds, GT-02287, for Parkinson’s Disease.
The study was conducted using human-induced pluripotent stem cells (iPSC) derived from donor patients with GBA-associated Parkinson’s disease, which were then differentiated into dopaminergic neurons.
GT-02287 demonstrated statistically significant, positive effects across a range of measurable parameters, including an increase in GCase levels and lysosomal transport.
It also reduced pathologically elevated levels of phosphorylated and aggregated α-synuclein, which are known to be a critical marker of Parkinson’s-induced neurodegeneration.
The preclinical data demonstrate that GT-02287 stabilizes and restores the function of misfolded GCase, thus correcting a prominent enzymatic dysfunction underlying GBA1 Parkinson’s disease pathology.
Approximately 14% of Parkinson’s patients are known to possess a mutation in the GBA1 gene, which encodes for the lysosomal enzyme GCase, and represents the most common genetic risk factor for Parkinson’s disease.
Gain believes that GT-02287, with its ability to restore GCase enzyme function, may be able to protect against, or possibly reverse, the damaging cascade of neuronal degeneration.
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