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…

STAR Candidates Boost GCase in Cell, Animal Models

Gain Therapeutics‘ investigational compounds were able to lessen motor symptoms in a rat model of GBA1-associated Parkinson’s disease and also decrease the levels of alpha-synuclein accumulation in nerve cells — two hallmarks of the neurodegenerative disease. The two candidates — GT-02287 and GT-02329 — are…