BETHESDA, Md., May 06, 2022 (GLOBE NEWSWIRE) — Gain Therapeutics, Inc. (GANX) (“Gain” or the “Company”), a biotechnology company applying its proprietary computational discovery platform to identify novel targets and small molecular treatments, announced today that it has presented positive preclinical data from its GBA Parkinson’s Disease (PD) program. The findings were highlighted in a poster presentation at the XXVII World Congress on Parkinson’s Disease and Related Disorders (IAPRD) held May 1-4, 2022 in Prague, Czech Republic. The data demonstrates the ability of lead compound GT-02287 to enter the brain, target and bind to beta-glucocerebrosidase (GCase) protein, preventing its degradation and allowing its transport to the lysosomes where the enzyme can exert its biological function.
“The data we presented at IAPRD further supports the therapeutic potential of our lead compound to alter the course of historically challenging neurodegenerative diseases,” said Eric Richman, CEO of Gain. “The disease-modifying potential of this compound may have a profound effect on patients suffering from Parkinson’s disease.”
Dr Manolo Bellotto, Chief Strategy Officer, said: “We continue to focus on targeting the cause of the disease, rather than its symptoms, and the data presented today continues to demonstrate that improving Lysosomal GCase activity by GT-02287 protects against major pathological features of PD, including alpha-synuclein-mediated pathology, enhancing the dopamine cell phenotype. We look forward to sharing additional findings as we continue to advance GT-02287 into the clinic this year. »
Data was generated in two different in vitro models and one in vivo model. The in vitro models used were the rotenone model in SH-SY5Y dopaminergic neurons and the CBE model in cortical neurons, both used to model disease pathology. The in vivo data was produced using the rotenone model in rats, which causes increased aggregated alpha-synuclein and reduced levels of dopamine in the striatum, two well-known markers of disease.
The presentation entitled “GT-02287, a structurally targeted brain-penetrating allosteric regulator for glucocerebrosidase shows evidence of pharmacological efficacy in models of Parkinson’s disease” showed that the orally bioavailable and brain-penetrating lead molecule had a promising effect, including:
- Compound penetration into brain and plasma
- Reduced P129-alpha-synuclein levels in SH-SY5Y dopaminergic neurons after rotenone injury
- Enhanced alpha-synuclein pathology in TH+ neurons after EPC injury
- Reduced aggregate alpha-synuclein in the substantia nigra and increased dopamine levels in the striatum in rotenone-injured rats
A copy of the poster is available on the Gain Therapeutics website at https://www.gaintherapeutics.com.
About Parkinson’s disease
Mutations in the GBA1 gene, encoding the lysosomal enzyme GCase, represent the most common genetic risk factor for PD. Impaired GCase function has attracted attention due to its association with α-synuclein pathology in patients with ACS-associated PD, but also in sporadic PD, as well as in associated α-synucleopathies. Although less studied, the decrease in GCase levels and activity is also implicated in the pathophysiology of AD. Enhancing mutant and wild-type GCase activity may represent a therapeutic strategy for the treatment of neurodegenerative diseases.
About Gain Therapeutics, Inc.
Gain Therapeutics, Inc. is transforming the drug discovery paradigm with structurally targeted allosteric regulators identified with its proprietary SEE-Tx® computational discovery platform. The ability to identify novel allosteric targets on proteins implicated in disease across therapeutic areas provides opportunities for a range of drug-protein interactions, including protein stabilization, protein destabilization, degradation protein targeting, allosteric inhibition and allosteric activation. Gain’s pipeline covers neurodegenerative diseases, lysosomal storage disorders, metabolic diseases and oncology. Gain’s primary program in Parkinson’s disease has received financial support from the Michael J. Fox Foundation for Parkinson’s Disease Research (MJFF) and the Silverstein Foundation for Parkinson’s Disease with ACS, as well as the joint Eurostars-2 with co-funding from the European Union Research Union Horizon 2020 and Innosuisse. For more information, please visit https://www.gaintherapeutics.com.
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