Study results demonstrate lead compounds increase GCase protein levels, activity and co-localization in the lysosome, and decrease toxic substrate accumulation
BETHESDA, Md., March 29, 2022 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”), a biotechnology company transforming the drug discovery paradigm with structurally targeted allosteric regulators identified with its proprietary computational discovery platform, today presented new pre-clinical data from its Gaucher Disease (GD) program. The results were highlighted in a poster presentation at the 2022 Glycolipid and Sphingolipid Biology GRC being held March 27 – April 1, 2022, in Castelvecchio Pascoli, Italy. The data show that the tested compounds increase the levels and activity of the beta-glucocerebrosidase (GCase) protein, increase co-localization of GCase with lysosomes, and most importantly, decrease disease-causing toxic substrate accumulation in normal and in patient-derived cortical neurons.
“The data presented today provides further confirmation of the effect of our lead compound in the Gaucher Disease program,” said Eric Richman, Chief Executive Officer of Gain. “This structurally targeted allosteric regulator can cross the blood-brain barrier and may have disease-modifying potential for patients with neuronopathic Gaucher disease who are currently without effective treatment options.”
The data was generated in two different in vitro models using normal, or wild-type (WT) human cortical neurons and cortical neurons derived from induced pluripotent stem cells (iPSCs) of Gaucher patients. The presentation titled “Structurally Targeted Allosteric Regulators Show Promising Therapeutic Effect in Gaucher Disease Cortical Neurons” showed that the orally bioavailable and brain-penetrant lead molecules have a promising effect, including:
- Significant increase of GCase levels and activity in WT and GD cortical neurons.
- Increase of lysosomal GCase levels in WT and GD cortical neurons.
- Effective depletion of toxic substrate levels in GD cortical neurons and GD patient-derived fibroblasts.
About Gaucher Disease
Gaucher disease is an inherited lysosomal storage disease caused by homozygous mutations of the GBA1 gene that result in the misfolding and subsequent dysfunction of beta-glucocerebrosidase (GCase), an enzyme that breaks down fatty chemicals in the body, including glucosylceramide and glucosylsphingosine. Partial or complete loss of GCase activity can cause the buildup of glucosylceramide and glucosylsphingosine in the lysosomes of macrophages, and the accumulation of these lipid substrates within the CNS can damage neuronal cells and result in neurological symptoms. Gaucher disease is traditionally classified according to one of three types, of which Gaucher disease type 1 is a non-neuronopathic form, whereas Gaucher disease types 2 and 3 are considered neuronopathic Gaucher disease (nGD) with early onset brain degeneration that gets progressively worse over time. There are no available treatment options for nGD because current enzyme replacement therapy does not cross the blood-brain barrier and is not efficient in treating neurological manifestations, therefore creating a significant unmet medical need in this patient population.
About Gain Therapeutics, Inc.
Gain Therapeutics, Inc. is transforming the drug discovery paradigm with structurally targeted allosteric regulators identified with its proprietary computational discovery platform SEE-Tx®. 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, metabolic diseases and 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 https://www.gaintherapeutics.com.
Any statements in this release that are not historical facts may be considered to be “forward-looking statements.” Forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties which may cause results to differ materially and adversely from the statements contained herein. Such statements include, but are not limited to, the expected progress of research and development programs and the effect of structurally targeted allosteric regulators for the treatment of Gaucher disease. Some of the potential risks and uncertainties that could cause actual results to differ from those expected include Gain’s ability to: make commercially available its products and technologies in a timely manner or at all; enter into strategic alliances, including arrangements for the development and distribution of its products; obtain intellectual property protection for its assets; accurately estimate and manage its expenses and cash burn and raise additional funds when necessary. Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made. Except as required by law, Gain does not undertake any obligation to update any forward-looking statements to reflect new information, events or circumstances after the date they are made, or to reflect the occurrence of unanticipated events.
Joleen Schultz & Associates