GBA-targeted therapy
GCase activators and substrate-reduction therapies aimed at GBA-mutation carriers (ambroxol, venglustat, etc.).
State of the art
No update yet for GBA-targeted therapy. An update is a standalone state-of-the-art for the topic — what someone with Parkinson's needs to know about where this approach stands today.
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With new FDA green light for developer, Parkinson’s clinical trial to launch soon
GT-02287, an oral GCase activator from Gain Therapeutics, received FDA IND clearance to start a Phase 2 trial (July–September 2026, sites in the US, Australia, and Europe). Earlier open-label Phase 1b data showed improved motor function, daily living, gait, sleep, and smell over three months, plus an 81% average reduction in the biomarker GluSph in patients with elevated baseline levels.