The Human Research Ethics Committee (HREC) in Australia cleared Gain Therapeutics to begin a Phase 1 trial of GT-02287, an oral compound in development for the treatment of GBA-1 Parkinson’s disease.
GT-02287 targets glucocerebrosidase (GCase), a protein that’s encoded by the GBA1 gene. Mutations in the GBA1 gene, which cause the misfolding of GCase, are a well-known genetic risk factor for the development of Parkinson’s disease and other synucleinopathies. GCase, known as a “housekeeping enzyme,” allows lysosomes to break down toxins and molecular waste, including aggregates of misfolded alpha-synuclein.
Gain uses their Site-Directed Enzyme Enhancement Therapy (SEE-Tx®) platform to analyze the 3D structure of proteins to identify new treatment targets in neurodegenerative disorders. Previous research demonstrated that GT-02287 was able to guide misfolded GCase into its proper shape and restore its activity.
“This approval is a key step for Gain and further validates our differentiated allosteric approach and our computational drug discovery platform as we endeavor to bring a novel, potentially disease-modifying therapeutic to patients with GBA1-Parkinson’s disease,” said Matthias Alder, Chief Executive Officer of Gain Therapeutics.
Gain did not provide specifics but said that a Phase 1 trial investigating the pharmacokinetics and safety of GT-02287 is expected to begin soon.