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Descriere companie
Gain Therapeutics, Inc (www.gaintherapeutics.com). is a holding company. The Company, through its subsidiary, GT Gain Therapeutics SA, is a development stage biotechnology company. The Company is focused on developing therapeutics to treat diseases caused by protein misfolding, with an initial focus on lysosomal storage disorders (LSDs), including rare genetic diseases and neurological disorders. It uses its platform, Site-Directed Enzyme Enhancement Therapy (SEE-Tx), to discover allosteric sites on misfolded proteins and identify proprietary small molecules that bind these sites and restore protein folding, potentially treating the underlying disease. Its pipeline includes its Structurally Targeted Allosteric Regulators (STARs), which are small molecules with the potential to treat diseases with unmet medical need. These diseases include Morquio B, GM1 Gangliosidosis (GM1), neuronopathic Gaucher disease, GBA1 Parkinson’s disease, Krabbe disease and Mucopolysaccharidosis type 1.