Gain Therapeutics presents data at AD/PD 2024 conference

Gain Therapeutics announces the presentation of a poster at the International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders, AD/PD 2024, that addresses the mechanism of action of the Company’s lead compound, GT-02287. The poster, titled “GT-02287, a Clinical Stage Glucocerebrosidase Regulator for the Treatment of PD, Eases ER Stress and Enhances Lysosomal Enzyme Activity,” which will be available online March 5th and presented on-site on the 8th and 9th by Dr. Natalia Perez-Carmona, demonstrates how GT-02287, through its interaction with glucocerebrosidase in the endoplasmic reticulum, aids correct GCase folding, preventing ER retention, ER stress, and ER-associated degradation of mutated GCase enzyme. GCase is consequently able to travel to the lysosome, resulting in enhanced lysosomal activity and efficient processing of the GCase substrate glucosylceramide. Increase in GCase substrate in the lysosome was previously shown to be associated with accumulation of aggregated alpha-synuclein, a pathological hallmark of Parkinson’s disease and related disorders.