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Gain Therapeutics Presents New Preclinical Data Demonstrating a Reduction of Plasma Neurodegeneration Biomarker NfL after Administration of its Drug Candidate GT-02287 in GBA1 Parkinson’s Disease Model
Treatment with GT-02287 also restored glucocerebrosidase (GCase) enzymatic function, reduced aggregated α-synuclein, neuroinflammation and neuronal death,
About this update from Gain Therapeutics, Inc.
[{"type":"text","content":"Treatment with GT-02287 also restored glucocerebrosidase (GCase) enzymatic function, reduced aggregated α-synuclein, neuroinflammation and neuronal death, increased dopamine levels and improved motor function Data presented at the International Congress of Parkinson's Disease and Movement Disorders® Gain remains on track to begin a Phase 1 clinical study of GT-02287 later this year BETHESDA, Md., Aug. 28, 2023 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today announced preclinical data demonstrating the Company’s lead drug candidate GT-02287 significantly decreased Parkinson’s disease (PD)-associated pathology and improved motor dysfunction in two different preclinical models of Parkinson’s disease. Of note, GT-02287 significantly reduced plasma Neurofilament Light Chain (NfL) levels, an emerging biomarker for neurodegeneration, in a mouse model of GBA1-PD. The findings are being presented in two posters, including one that was accepted as a late breaking abstract, at the International Congress of Parkinson's Disease and Movement Disorders® being held in Copenhagen, Denmark from August 27-31, 2023. “The compelling data from two preclinical Parkinson’s disease models with our lead drug candidate GT-02287 show that this orally administered allosteric regulator of GCase restored enzymatic function and significantly reduced the pathological hallmarks and motor dysfunction associated with Parkinson’s disease,” said Joanne Taylor, Ph.D., Senior Vice President of Research at Gain. “GT-02287 also markedly decreased plasma NfL, an emerging and important neurodegeneration biomarker that can be measured in blood, which is very exciting as we consider its application in clinical trials.” The first poster titled “Neuroprotective effect of GT-02287, a brain-penetrant structurally targeted allosteric regulator of glucocerebrosidase, leads to a significant reduction of plasma NfL levels and improvement in behavioural deficits in a mouse model of GBA1 Parkinson’s disease” (Late-breaker Abstract #LBA-13) will be presented by Dr. Taylor on each poster presentation date between 13:00 and 15:00 in the exhibition hall. Researchers used a mouse model of GBA1-PD, created using conduritol...