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Gain Therapeutics Presents New Preclinical Data from its Gaucher Disease Program at the 19th Annual WORLDSymposium

Study results support the disease-modifying potential of GCase-targeting small molecule therapy for neuronopathic Gaucher disease

BETHESDA, Md., Feb. 27, 2023 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a biotechnology company transforming drug discovery with its proprietary computational discovery platform identifying novel allosteric binding sites and creating small molecule treatments, presented new pre-clinical data from its Gaucher disease program in a poster presentation at the 19th Annual WORLDSymposium held February 22–26, 2023, in Orlando, Florida. The data generated in an animal model of neuronopathic Gaucher disease (nGD) show that GT-02329 restores β-glucocerebrosidase (GCase) activity, depletes accumulation of toxic lipid substrates, reduces neuroinflammation and improves neuromuscular function.

“These data are a further validation of the disease-modifying potential of our GCase-targeting small molecule compounds in diseases caused by mutations of the GBA1 gene, including for the treatment of nGD and GBA1 Parkinson’s disease,” said Matthias Alder, Chief Executive Officer of Gain. “The consistency of the extensive preclinical data package we have generated with our GCase-targeting drug candidates is encouraging, and we remain on track to submit the dossier for the start of the Phase 1 clinical study with our lead GCase-targeting compound GT-02287 in mid-2023.”

The effect of Gain’s small molecule structurally targeted allosteric regulators of GCase that act on lysosomal function was shown in studies conducted in the conduritol beta epoxide (CBE) mouse model. CBE is a covalent inhibitor that causes a reduction in GCase activity by reacting with the catalytic site of GCase and inactivating the GCase enzyme, which results in neurological features that occur in nGD patients, including the accumulation of the toxic lipid substrate glucosylsphingosine. The data presented in the poster titled “GT-02329, a structurally targeted allosteric regulator of GCase, restores GCase activity, reduces microgliosis and improves fine locomotor skills in the CBE model of neuronopathic Gaucher’s disease” demonstrated that: GT-02329 is orally bioavailable and brain penetrant, restored GCase activity, reduced accumulation of GCase substrates glucosylceramide and glucosylsphingosine, reduced a marker of inflammation (Iba-1), and improved neuromuscular function in a dose-dependent manner in CBE-injured mice treated with the compound.

A PDF of the poster presented at the WORLDSymposium is available on the Science & Technology section of the Company’s website at .

About Gaucher Disease

Gaucher disease (GD) is a lysosomal storage disease characterized by deficiency in the β-glucocerebrosidase (GCase) enzyme encoded by mutated forms of the GBA1 gene. This causes the accumulation of toxic lipid substrates in the liver, spleen and bone marrow and, in neuronopathic GD (nGD), also in the nervous system. Current standard of care for GD, namely enzyme replacement therapy, does not cross the blood-brain barrier, leaving an unmet medical need for the development of novel therapies for nGD patients. Gain Therapeutics has applied its proprietary computational drug discovery platform, Site-directed Enzyme Enhancement Therapy (SEE-Tx®), to the development of small-molecule structurally targeted allosteric regulators (STARs) that bind to GCase, stabilize it, and restore its function. SEE-Tx® is a fast and cost-effective solution that has allowed us to discover STARs of the GCase enzyme that are orally bioavailable and brain-penetrant.

About Gain Therapeutics, Inc.

Gain Therapeutics, Inc. is transforming drug discovery with its proprietary computational discovery platform identifying novel allosteric binding sites and creating small molecule treatments to address unmet medical needs. The ability to identify never-seen-before allosteric targets on proteins involved in diseases across the full spectrum of therapeutic areas provides 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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