Gain readies for Phase 1b trial of Parkinson’s therapy GT-02287

Gain Therapeutics has developed an oral small molecule called GT-02287, which was found to be safe and well tolerated in healthy volunteers during a Phase 1 clinical trial. Importantly, it was able to reach the brain, showing promise as a treatment for Parkinson’s disease linked to mutations in the GBA gene. This gene is responsible for producing an enzyme that breaks down certain fats in cells, and when it malfunctions, harmful proteins can build up in the brain, contributing to Parkinson’s. The company plans to start a Phase 1b trial in Parkinson’s patients by the end of the year to further assess the safety of GT-02287 and confirm its effectiveness. Early results are promising, and data from Parkinson’s patients are expected by mid-2025.