Gain Therapeutics' Gaucher, Parkinson's Candidate Shows Potential In Patient-Derived iPSC Study
- The topline data from a study conducted at the University of Maryland School of Medicine (UMSOM) by Gain Therapeutics Inc (NASDAQ: GANX) evaluating two lead Structurally Targeted Allosteric Regulators (STARs) compounds, GT-02287 and GT-02329, for Gaucher and GBA1 Parkinson's Disease, demonstrated positive effects on all tested phenotypes.
- The study used human induced pluripotent stem cells (iPSC) derived from GD and GBA-associated Parkinson's disease patients.
- Data showed that the compounds increased the levels of GCase protein, its transport to the lysosome, and its enzymatic activity.
- In dopaminergic neurons, the two lead STAR chaperones also decrease the levels of α-synuclein-p129, demonstrating their potential to treat GBA1-associated Parkinson's Disease.
- The Company anticipates initiating IND-enabling studies for Gaucher / Parkinson's Disease in Q4 of 2021.
- Price Action: GANX stock is up 11.14% at $8.78 during the market session on the last check Wednesday.
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