STAR Molecule to Restore GCase Activity Showing Early Potential

An oral candidate therapy for Parkinson’s disease called GT-02287 lowered the levels of alpha-synuclein accumulation and inflammation, and lessened behavioral deficits in animal models of the disease. “We are working diligently to advance this breakthrough compound to the clinic and bring it one step closer to meeting the currently unaddressed needs…

STAR Compounds Show Potential to Treat GBA1-linked Parkinson’s

Gain Therapeutics announced that preclinical studies with two of its compounds, GT-02287 and GT-02329, showed they could reduce the alpha-synuclein aggregates that characterize Parkinson’s and increase glucocerebrosidase protein levels in cell models of Parkinson’s and Gaucher disease. GT-02287 and GT-02329 are known as STARs, which stands for structurally targeted…

Small Molecules, STARs Treat Cell Models of GBA1-linked Parkinson’s

Two investigational small molecules known as GT-02287 and GT-02329 lowered toxic protein levels in patient-derived cell models of Parkinson’s disease associated with a mutation in GBA1, their developer, Gain Therapeutics, announced. Gain is planning additional preclinical studies this year that could support applications to regulatory agencies allowing the compounds to…