Gain Therapeutics, Inc. is a biotechnology company. The Company is focused on developing novel small molecule therapeutics to treat diseases across several therapeutic areas, including, central nervous system (CNS) disorders, lysosomal storage disorders (LSDs), metabolic disorders, and other diseases that can be targeted through protein degradation, such as oncology. The Company’s drug discovery platform, Magellan discovers novel allosteric binding sites on proteins implicated in a disease and to identify proprietary small molecules that bind these sites to modulate protein function and treat the underlying cause of the disease. Its lead product candidate, GT-02287, is being developed for the treatment of GBA1 Parkinson’s disease, including restoration of glucocerebrosidase or GCase, function in the lysosome, reduction of toxic lipid substrates and toxic forms of alpha-synuclein, improved survival of dopaminergic neurons, and enhance in dopamine levels.