Gain Therapeutics, Inc., a biotechnology company, engages in developing various therapies to treat diseases caused by protein misfolding. It focuses on rare genetic diseases and neurological disorders. The company uses its Site-Directed Enzyme Enhancement Therapy platform to discover allosteric sites on misfolded proteins and identify proprietary small molecules that bind these sites, restore protein folding, and treat disease. It is developing structurally targeted allosteric regulator candidates to treat various diseases, including Morquio B, GM1 gangliosidosis (GM1), neuronopathic Gaucher disease, GBA1 Parkinson's, Krabbe, and Mucopolysaccharidosis type 1 diseases. The company was founded in 2017 and is based in Bethesda, Maryland.