Gain Therapeutics Inc

Gain Therapeutics, Inc. is a biotechnology company that develops novel small molecule therapeutics across multiple therapeutic areas, operating in Switzerland, Spain, the United States, and Australia. Its drug discovery platform, Magellan, identifies novel allosteric binding sites in a disease and proprietary small molecules that bind these sites to modulate protein function, aiming to treat the underlying cause of disease. The lead drug candidate, GT-02287, is in a Phase 1b study for the treatment of Parkinson's disease with or without a GBA1 mutation. Additional small molecule candidates are in discovery, research, and preclinical stages for dementia with Lewy bodies, Alzheimer's disease, Gaucher's disease, lysosomal storage disorders, metabolic disorders, and solid tumors. The company was founded in 2017 and is based in Bethesda, Maryland.

Biotech & Genomic Medicine▲

Gain Therapeutics Updates Parkinson's Pipeline, Promotes Executives, Extends Cash Runway Into Q2 2027

Gain Therapeutics provided a pipeline update, leadership promotions, and cash position outlook. Lead Parkinson's candidate Rexaceract maintained a favorable safety profile through five months in its Phase 1b trial, with results expected in the fourth quarter of 2026. The company reported an 81% average reduction in cerebrospinal fluid GluSph levels after 90 days in patients with elevated baseline levels, and a 4.8-point improvement in MDS-UPDRS scores after 150 days. The FDA authorized an IND in the second quarter of 2026, enabling a Phase 2 trial launch by the third quarter. Gain also promoted Joanne Taylor to Chief Scientific Officer and Terenzio Ignoni to Chief Operating Officer, and reported $16.5 million in cash as of March 31, 2026, funding operations into the second quarter of 2027.

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Biotech & Genomic Medicine

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