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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

BETHESDA, 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 hippocampal neurons challenged with Aβ-1-42 and human Tau oligomers (hTauO), respectively. The data presented in the poster titled “Small-Molecule Structurally Targeted Allosteric Regulators of Glucocerebrosidase Show Neuroprotective Properties in Cell-Based Models of Alzheimer’s Disease,” demonstrate in vitro evidence that a STAR compound improves neuronal survival and neurite network and reduces hyperphosphorylated tau in primary rat cortical neurons after Aβ-1-42 injury. Additionally, STAR compounds were shown to reduce hTauO-induced neurotoxicity in primary rat hippocampal neurons. The data therefore demonstrate that structurally targeted allosteric regulators are a potential disease-modifying, novel pharmacological option for the treatment of AD and other tauopathies that warrants further investigation.

A PDF of the poster presented at the AD/PD 2023 Annual Meeting is available on the Science & Technology section of the Company’s website at .

About Gain Therapeutics, Inc.

Gain Therapeutics, Inc. is a biotechnology company leading the discovery and development of allosteric small molecule therapies. With its proprietary computational discovery platform SEE-Tx®, Gain Therapeutics is transforming drug discovery by identifying novel allosteric targets on proteins involved in diseases across the full spectrum of therapeutic areas. By binding to allosteric binding sites, the small molecules discovered with SEE-Tx provide opportunities for a range of drug-protein interactions, including protein stabilization, protein destabilization, targeted protein degradation, allosteric inhibition, and allosteric activation. Gain’s pipeline spans neurodegenerative diseases, lysosomal storage disorders (LSDs), metabolic disorders, as well as other diseases that can be targeted through protein degradation, such as oncology. Gain’s lead program in Parkinson’s disease has been awarded funding support from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and The Silverstein Foundation for Parkinson’s with GBA, as well as from the Eurostars-2 joint program with co-funding from the European Union Horizon 2020 research and Innosuisse. For more information, please visit 

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