GT-02287 is a drug under development by Gain Therapeutics that targets misfolded proteins, specifically glucocerebrosidase (GCase) proteins, in conditions like Dementia with Lewy bodies and Alzheimer’s disease. The drug is a small molecule that works by stabilizing and enhancing the function of the GCase enzyme, which is important for breaking down and removing certain lipids in the brain. In Parkinson’s disease, mutations in the GBA1 gene, which encodes the GCase enzyme, lead to a reduction in enzyme activity, resulting in the accumulation of toxic substances like glucosylceramide. GT-02287 is designed to bind to the misfolded GCase, stabilizing its structure and improving its function, thereby reducing the accumulation of these toxic substances. This mechanism is thought to alleviate some of the cellular dysfunctions associated with Alzheimer’s Disease and the accumulation of Lewy bodies and potentially slow disease progression.