Core Insights - Gain Therapeutics, Inc. has formed a Clinical Advisory Board (CAB) to support the development of its lead drug candidate, GT-02287, for Parkinson's disease [1][2] - The CAB consists of experts in trial design, genetics, and biomarkers related to Parkinson's disease, aiming to advance GT-02287 through Phase 2 and Phase 3 clinical development [1][2] Company Overview - Gain Therapeutics is a clinical-stage biotechnology company focused on discovering and developing next-generation allosteric small molecule therapies [9][10] - The lead drug candidate, GT-02287, is being evaluated for treating Parkinson's disease with or without a GBA1 mutation [9][10] Drug Candidate Details - GT-02287 is an orally administered, brain-penetrant small molecule that acts as an allosteric protein modulator, restoring the function of the lysosomal enzyme glucocerebrosidase (GCase) [6][9] - Preclinical studies have shown that GT-02287 can restore GCase function, reduce aggregated α-synuclein, and improve motor and cognitive functions [6][7] Clinical Development and Support - The drug candidate has received funding support from The Michael J. Fox Foundation for Parkinson's Research and The Silverstein Foundation for Parkinson's with GBA, among others [8] - Results from a Phase 1 study indicated favorable safety and tolerability, with plasma exposure in the projected therapeutic range [9]
Gain Therapeutics Announces Formation of Clinical Advisory Board to Support Continued Advancement of Lead Drug Candidate GT-02287