Does anyone have any knowledge of the work being done by Gain Therapeutics on GT-02287 a molecule which penetrates the BBB and is claimed to improve the lot of all PwPs (fairly dramatically).
it is designed to restore lysosomal enzyme GCase function but is said to help anyone whether or not you have the GBA1 mutation. It is said to have the ability to halt the progression of PD. This seems to be potentially life changing for all of us. Am I being unduly optimistic or is this the game changer we have been waiting for?
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Lyricist
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Gain Therapeutics’ lead drug candidate, GT-02287, is in clinical development for the treatment of Parkinson’s disease (PD) with or without a GBA1 mutation. The orally administered, brain-penetrant small molecule is an allosteric protein modulator that restores the function of the lysosomal protein enzyme glucocerebrosidase (GCase) which becomes misfolded and impaired due to mutations in the GBA1 gene, the most common genetic abnormality associated with PD, or other age-related stress factors. In preclinical models of PD, GT-02287 restored GCase enzymatic function, reduced aggregated α-synuclein, neuroinflammation and neuronal death, and improved motor function and cognitive performance. Additionally, GT-02287 significantly reduced plasma neurofilament light chain (NfL) levels, an emerging biomarker for neurodegeneration.
Compelling preclinical data in models of both GBA1-PD and idiopathic PD, demonstrating a disease-modifying effect after administration of GT-02287, suggests that GT-02287 may have the potential to slow or stop the progression of Parkinson’s disease."
"Review of unblinded data after database lock confirmed that single and multiple doses of GT-02287 were safe and generally well tolerated up to and including the highest planned dose levels across all age groups (approximately 15% of which were over the age of 50 years). GT-02287 was present in the cerebrospinal fluid (CSF) and peripheral target engagement was demonstrated. The favorable safety and tolerability profile at oral dose levels that resulted in therapeutic plasma levels, CNS exposure, and target engagement further strengthens GT-02287’s potential to be a lead treatment for Parkinson’s disease in patients with or without a GBA1 mutation.
“On the heels of this data, we expect to initiate a trial in people with Parkinson’s disease by Q4 2024 with the goal of demonstrating safety and tolerability in patients with Parkinson disease and to obtain proof of mechanism based on relevant biomarkers. We anticipate having data from Parkinson’s disease patients by mid-point 2025,” commented Jonas Hannestad, M.D., Ph.D., Chief Medical Officer of Gain."
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