Core Insights - Gain Therapeutics, Inc. is presenting a poster on the Phase 1b clinical trial design for its drug candidate GT-02287 aimed at treating Parkinson's disease at the AD/PD™ 2025 conference [1][2] Company Overview - Gain Therapeutics is a clinical-stage biotechnology company focused on developing next-generation allosteric small molecule therapies [5] - The lead drug candidate, GT-02287, is designed to treat Parkinson's disease with or without a GBA1 mutation and is an orally administered, brain-penetrant small molecule [2][5] Drug Candidate Details - GT-02287 functions as an allosteric enzyme modulator that restores the function of the lysosomal enzyme glucocerebrosidase (GCase), which is often impaired due to GBA1 gene mutations [2] - Preclinical studies have shown that GT-02287 can restore GCase function, reduce aggregated α-synuclein, decrease neuroinflammation and neuronal death, and improve both motor and cognitive functions [2][3] - The drug has also been noted to significantly lower plasma neurofilament light chain (NfL) levels, a biomarker for neurodegeneration [2] Clinical Development - The Phase 1 study of GT-02287 in healthy volunteers demonstrated favorable safety, tolerability, and target engagement with the GCase enzyme [5] - Compelling preclinical data suggest that GT-02287 may have a disease-modifying effect, potentially slowing or stopping the progression of Parkinson's disease [3] Funding and Support - Gain Therapeutics has received funding support from The Michael J. Fox Foundation for Parkinson's Research, The Silverstein Foundation for Parkinson's with GBA, and the Eurostars-2 program co-funded by the European Union Horizon 2020 research and Innosuisse [4]
Gain Therapeutics Announces Poster Presentation at AD/PD 2025