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Gain Therapeutics develops therapies for diseases caused by protein misfolding, focusing on rare genetic and neurological disorders using its Site-Directed Enzyme Enhancement platform to identify small molecules that restore protein function.

Gain Therapeutics' lead candidate, GT-02287, an oral small molecule in clinical trials for Parkinson's, aims to restore GCase enzyme function in GBA1 mutation patients as a potential disease-modifying therapy.
Overall rating: C
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