BETHESDA, Md. , May 11, 2022 (GLOBE NEWSWIRE) – Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a biotech company that is transforming drug discovery with its computational discovery platform identifying new allogeneic binding sites and creating small molecule therapies, today provided additional confirmatory data from its program Gaucher disease. The results are highlighted in an oral presentation at 1St IWGDD Symposium 8-11 May 2022 in the Netherlands. The data demonstrate that the tested compounds increase the levels and activity of the protein beta-glucocerebrosidase (GCase), increase the localization of GCase with lysosomes, and most importantly, reduce the accumulation of a pathogenic toxic substrate in patient-derived and transgenic fibroblasts. Mouse model of Gaucher disease.
“At Gain, we have been applying the power and precision of our See-Tx platform to develop pharmacological chaperones, which are small molecules capable of crossing the blood-brain barrier, to prevent GCase protein disruption and restore enzymatic function within the brain,” said Eric Richman, CEO of Gain Therapeutics. “The data we shared today support GT-02329 as a potential treatment to mitigate the neurological effects of GD, and we look forward to exploring the benefits of this treatment for patients in need of further advances as we advance this compound in the clinic.”
Data were generated in two different in vitro models in wild-type (WT) patient-derived fibroblasts and in patient-derived dopaminergic neurons, as well as in an in vivo transgenic mouse model harboring the L444P mutation. The presentation titled “Structurally-targeted Allosteric Regulators of Glucocerebrosidase (GCase) Show Promising Pharmacological Activity in L444P Models of Gaucher Disease”, demonstrated that an orally bioavailable brain-penetrating lead molecule has a promising effect, including:
- Increased GCase activity in WT and GD patient-derived fibroblasts via a number of dominant mutations
- Significant decrease in GlcCer in the lysosome in GD patient-derived fibroblasts
- Significant increase in plasma and brainstem GCase activity and decrease in plasma GlcSph in a transgenic mouse model harboring the L444P mutation
A copy of the presentation is available on the Gain Therapeutics website at https://www.gaintherapeutics.com.
About Gaucher disease
Gaucher disease is an inherited disease related to lysosomal storage caused by homozygous mutations in the GBA1 gene that results in modulation and subsequent dysfunction of beta-glucocerebrosidase (GCase), an enzyme that breaks down lipid chemicals in the body, including glucosylceramide and glucosylphingosine. Partial or complete loss of GCase activity can cause the accumulation of glucosylceramide and glucosyl sphingosine in lysosomes in macrophages, and the accumulation of these lipid substrates within the CNS can damage neurons and lead to neurological symptoms. Gaucher disease is traditionally classified according to one of three types, of which type 1 Gaucher disease is a non-neuropathy form, while Gaucher disease type 2 and 3 is neurological Gaucher disease (nGD) with progressively worsening early onset of cerebral degeneration. the time. There are no available treatment options for nGD because current enzyme replacement therapy does not cross the blood-brain barrier and is ineffective in treating neurological manifestations, thus creating a significant unmet medical need in these patients.
About Gain Therapeutics, Inc.
Gain Therapeutics, Inc. transforms the drug discovery paradigm using structurally targeted allosteric regulators identified through its SEE-Tx® computational discovery platform. The ability to identify previously unparalleled allogenic targets on proteins implicated in diseases across a full range of therapeutic domains provides opportunities for a range of drug-protein interactions, including protein stabilization, protein destabilization, target protein degradation, allosteric inhibition and allosteric activation. Gain’s pipeline includes neurodegenerative diseases, lysosomal storage disorders, metabolic diseases, and tumors. Gain’s pioneering program in Parkinson’s disease has received funding support from the Michael J. Fox Foundation for Parkinson’s Research (MJFF) and the Silverstein Foundation for Parkinson’s Disease with GBA, as well as from the joint Eurostars-2 program co-funded by the European Union Research Union Horizon 2020 and Innosuisse. For more information, please visit the website https://www.gaintherapeutics.com.
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