Bethesda, MD, August 29, 2023 (PlatoData via 500NewsWire) — Gain Therapeutics, Inc. (Nasdaq: GANX), a pioneering biotechnology company specializing in innovative allosteric small molecule therapies, has made significant strides in the fight against Parkinson's disease (PD). The Company's lead drug candidate, GT-02287, has demonstrated extraordinary potential in mitigating the effects of Parkinson's disease across two distinct preclinical models of the ailment. These findings suggest that GT-02287 has the capacity to alleviate Parkinson's disease pathology and enhance motor function.
A notable highlight is the substantial reduction of plasma Neurofilament Light Chain (NfL) levels, an emerging biomarker for neurodegeneration. This advancement was demonstrated in a mouse model of GBA1-PD, a discovery that could have far-reaching implications for understanding neurodegenerative conditions.
These groundbreaking findings were presented at the International Congress of Parkinson's Disease and Movement Disorders, held in Copenhagen, Denmark from August 27 to 31, 2023.
Dr. Joanne Taylor, Senior Vice President of Research at Gain, expressed her excitement, stating, "The data from these preclinical models highlight GT-02287's potential as an oral allosteric regulator of GCase. The restoration of enzymatic function and reduction in Parkinson's disease-related pathologies are promising indicators. Additionally, the significant reduction in plasma NfL levels, a key neurodegeneration biomarker, holds promise for future clinical trials."
Of significant importance is the substantial decrease in plasma NfL levels observed in the GBA1-PD model, signifying a potential advancement in neurodegeneration assessment. The second poster, "GT-02287, a brain-penetrant structurally targeted allosteric regulator," scheduled for August 30th, will delve into GT-02287's impact on a PD animal model, showcasing its potential to restore protein function and counteract neurotoxic effects.
Matthias Alder, Chief Executive Officer of Gain Therapeutics, emphasized the significance of these results. He stated, "The data from both posters solidify the potential of GT-02287 to restore GCase protein function, shield against Parkinson's disease's neurological impact, and reduce neurodegeneration, as indicated by the emerging biomarker NfL. These compelling findings pave the way for Phase 1 clinical trials with GT-02287, marking a pivotal step towards advancing a potentially groundbreaking treatment for GBA1 Parkinson's disease." Gain Therapeutics' achievements underscore its pivotal role in shaping the landscape of Parkinson's disease research and treatment.
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Gain Therapeutics, Inc.