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Gain Therapeutics to Present Positive Preclinical Data on its GBA1 Program at the AD/PD 2023 Meeting
Study results support the disease-modifying potential of structurally targeted allosteric regulators of GCase for the treatment of Alzheimer’s disease
About this update from Gain Therapeutics, Inc.
[{"type":"text","content":"Study results support the disease-modifying potential of structurally targeted allosteric regulators of GCase for the treatment of Alzheimer’s disease\nBETHESDA, Md., March 29, 2023 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a biotechnology company leading the discovery and development of allosteric small molecule therapies, today announced the presentation of new pre-clinical data supporting the potential of its structurally targeted allosteric regulators (STARs) of glucocerebrosidase (GCase) to prevent neurodegeneration in Alzheimer’s disease (AD). The data will be shown in a poster presentation at the 2023 International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders (AD/PD) being held March 28 – April 1, 2023 in Gothenburg, Sweden. “We are pleased to expand upon the extensive package of preclinical data generated with our STAR compounds, which continue to demonstrate their potential advantages across a range of disorders associated with GCase protein misfolding,” said Matthias Alder, Chief Executive Officer. “We have shown previously that STAR-mediated augmentation of lysosomal GCase induces a neuroprotective effect in cellular and in vivo models of GBA1 Parkinson’s disease and Gaucher disease. In the presentation at AD/PD, we showcase the neuroprotective properties of these compounds in cellular models of Alzheimer’s disease, which support their potential for the treatment of Alzheimer disease and other tauopathies.” Dr. Joanne Taylor, SVP, Head of Research, added: “Augmenting and restoring GCase function appears to constitute a fundamental mechanism of action that can result in beneficial effects across a range of neurodegenerative disease pathologies and indications. We look forward to providing further updates on our allosteric regulator programs throughout the year.” The data generated in two cell-based assays of Alzheimer’s disease showed that Gain’s orally bioavailable, brain-penetrant structurally targeted allosteric regulators of GCase show promising activity against Amyloid Beta 1-42 (Aβ-1-42) and oligomeric Tau toxicity, which are thought to underlie neurodegeneration and cognitive impairment in Alzheimer's disease. The neuroprotective properties of STAR compounds were evaluated in cellular models of AD with primary rat cortical and ...