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Gain Therapeutics Presents Data at the AD/PD™ 2024 Conference Demonstrating the Mechanism of Action of GT-02287, its Clinical Stage GCase Regulator for the Treatment of Parkinson’s Disease
Data support the ability of GT-02287 to prevent several steps in the disease cascade resulting from GCase misfolding and dysfunction BETHESDA, Md., March 05,
About this update from Gain Therapeutics, Inc.
[{"type":"text","content":"Data support the ability of GT-02287 to prevent several steps in the disease cascade resulting from GCase misfolding and dysfunction\nBETHESDA, Md., March 05, 2024 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today 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. AD/PD™ 2024 is being held March 5th – 9th, 2024 in Lisbon, Portugal. 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 (GCase) in the endoplasmic reticulum (ER), 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. The study was done in collaboration with scientists in the group of Professor Maurizio Molinari at the Università dela Svizzera italiana-affiliated Institute for Research in Biomedicine in Bellinzona, Switzerland and was conducted under the support of a Eurostars-2 joint program. “These data further confirm our understanding of the mechanism of action of GT-02287, how it impacts cellular health by preventing the downstream consequences of GCase misfolding, including cellular stress and lysosomal dysfunction” commented Dr. Natalia Perez-Carmona, Senior Director of Biology at Gain Therapeutics and presenting author. “The AD/PD™ conference is an important opportunity for us to continue to discuss our differentiated approach to addressing GBA1 Parkinson’s disease through GCase modulation...