GT-02287and GT-02329 compounds display positive effects on GCase activity
and related parameters
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Study results demonstrate an increase in GCase protein levels with transport to the lysosomes and decrease of glucosylceramide and <ALPHA>-synuclein-p129 levels
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Comprehensive study results to be presented at Michael J. Fox Foundation's upcoming Innovating from Drug Discovery to the Clinic: Novel Approaches to PD Therapeutic Development webinar
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IND-initiating study planned for Q4'21
Gain Therapeutics, Inc. (Nasdaq: GANX) ("Gain", or the "Company"), a biotechnology company focused on identifying and optimizing allosteric binding sites never before targeted in neurodegenerative diseases and lysosomal storage disorders, today announced topline data from the Company's study, conducted at the University of Maryland School of Medicine (UMSOM). The study, evaluating two lead Structurally Targeted Allosteric Regulators (STARs) compounds -- GT-02287 and GT-02329 -- for the treatment of Gaucher and GBA1 Parkinson's Disease, demonstrated positive effects on all tested phenotypes.
Ricardo Feldman, Ph.D., Associate Professor of Microbiology and Immunology at the University of Maryland School of Medicine, stated, "Our laboratory is using human induced pluripotent stem cells (iPSC) derived from patients with GD and GBA-associated Parkinson's disease to test the efficacy of the two lead STAR chaperones developed by Gain Therapeutics. Our studies in iPSC-derived cortical and dopaminergic neurons from neuronopathic Gaucher Disease patients show that these compounds increase the levels of GCase protein, its transport to the lysosome, and its enzymatic activity. In dopaminergic neurons, the two lead STAR chaperones also decrease the levels of <ALPHA>-synuclein-p129, demonstrating their potential to treat GBA1-associated Parkinson's Disease."
"These data are extremely exciting, as it further demonstrates the potential of GT-02287 and GT-02329 and expands the body of evidence supporting our Site-Directed Enzyme Enhancement Therapy (SEE-Tx(TM)) drug discovery platform," said Eric Richman, Chief Executive Officer of Gain. "We plan to fully evaluate the results of this study and present a complete data set on the upcoming Michael J. Fox Foundation's Innovating from Drug Discovery to the Clinic: Novel Approaches to PD Therapeutic Development webinar. In addition, we anticipate initiating IND-enabling studies for Gaucher / Parkinson's Disease in the fourth quarter of this year."
Gain identified the two lead STAR candidates (GT-02287 and GT-02329) through its proprietary SEE-Tx(TM) platform. The compounds were characterized in assays to potentially help Parkinson's patients with GBA1 gene mutations as well as patients whose glucocerebrosidase (GCase) protein is misfolded due to ageing cellular processes. This previous work was supported through grants from The Michael J. Fox Foundation (MJFF) and the Silverstein Foundation for Parkinson's with GBA.
Mutations in GBA1, the gene encoding the lysosomal enzyme GCase, are among the most commonly known genetic risk factors for the development of Parkinson's Disease and related synucleinopathies. GBA1 mutations are causative for the rare autosomal storage disorder Gaucher Disease and may lead to degradation of the protein, disruptions in lysosomal targeting and diminished performance of the enzyme in the lysosome. Patients with Gaucher Disease including heterozygous carriers are at increased risk of developing Parkinson's Disease and Dementia with Lewy Bodies.
The study conducted in collaboration with Ricardo A. Feldman, Ph.D., Associate Professor of Microbiology and Immunology at UMSOM and lead investigator, is using human induced pluripotent stem cells (iPSC) for Gaucher and Parkinson's Disease modeling and drug discovery. Gain's candidate molecules GT-02287 and GT-02329 have been initially assessed in this unique "disease-in-a-dish" iPSC model derived from Gaucher type 2 and 3 patient cells.
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