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Gain Therapeutics, Inc. Announces Positive Topline Data in Patient-derived iPSC Study Evaluating STAR Compounds as a Treatment for Gaucher and Parkinson’s Disease

GT-02287and GT-02329 compounds display positive effects on GCase activity
and related parameters
Study results demonstrate an increase in GCase protein levels with transport to the lysosomes and decrease of glucosylceramide and α-synuclein-p129 levels
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
IND-initiating study planned for Q4’21

BETHESDA, Md., Sept. 08, 2021 (GLOBE NEWSWIRE) -- 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 α-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™) 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™ 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.

Topline data from collaboration with University of Maryland School of Medicine on GBA-associated Parkinson’s Disease in iPSC model. The two STAR compounds achieved the following results:

  • Displayed positive effects on key biological read-outs such as GCase activity, GCase protein levels, GCase transport to the lysosomes, glucosylceramide and depletion of P-alpha-synuclein: all parameters that are related to disease onset and progression
  • Increased GCase enzyme activity
  • Increased GCase protein levels and transport to the lysosome in cortical neurons
  • Decreased GluCer accumulation in cortical neurons
  • Increased GCase protein levels and transport to the lysosome in dopaminergic neurons
  • Decreased α-synuclein-p129 levels in dopaminergic neurons

“We appreciate the close collaboration with Dr. Feldman and the University of Maryland School of Medicine in this important area of research,” said Manolo Bellotto, Ph.D., General Manager at Gain. “The topline data demonstrates that our STARs compounds open a new potential approach for direct treatment of GBA1 Parkinson’s Disease by guiding misfolded forms of the GCase enzyme to their proper shape and restoring enzymatic activity. This is an exciting validation of our platform technology and a promising potential therapeutic opportunity for patients suffering from these debilitating diseases.”

The combined data from Gain Therapeutics and Dr. Feldman’s laboratory suggest that STARs bind to novel allosteric binding sites identified using Gain’s proprietary SEE-Tx™ drug discovery platform, stabilize, and restore enzymatic activity to misfolded GCase. In these studies, the restoration of GCase activity resulted in the depletion of the toxic phosphorylated and aggregated forms of α-synuclein and a reversal of the neurodegenerative process resulting in improved locomotor activity in Parkinson’s Disease model in rats. Given that STARs are small molecule therapies, they have significant advantages over the current standard of care for disorders associated with protein misfolding, including most enzyme replacement therapies and gene therapies. They can be administered as a simple oral pill and can reach organs and tissues not typically accessible through current therapeutic options, including the brain, bone and cartilage. As well, the Company believes that STARs are relatively easy and cost-effective to manufacture.

“These encouraging results show promise for this approach to correct dysfunction in the GBA1 pathway, a leading target for Parkinson’s drug development,” said Marco Baptista, PhD, MJFF Vice President of Research Programs. “We look forward to hearing more on next steps to advance these potential therapies further in testing and closer to patients whose greatest unmet need is a treatment to slow or stop disease progression.”

About Gain Therapeutics, Inc.
Gain Therapeutics, Inc. is positioned at the confluence of technology and healthcare and focused on redefining drug discovery with its SEE-Tx™ target identification platform. By identifying and optimizing allosteric binding sites that have never before been targeted, Gain is unlocking new treatment options for difficult-to-treat disorders characterized by protein misfolding. Gain was established in 2017 with the support of its founders and institutional investors. It 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. In July 2020, Gain Therapeutics, Inc. completed a share exchange with Gain Therapeutics, SA, a Swiss corporation, whereby GT Gain Therapeutics SA became a wholly owned subsidiary of Gain Therapeutics, Inc. For periods and at dates prior to the Corporate Reorganization, the consolidated financial statements were prepared based on the historical financial statements of GT Gain Therapeutics SA.

For more information, please visit

About the University of Maryland School of Medicine
Now in its third century, the University of Maryland School of Medicine was chartered in 1807 as the first public medical school in the United States. It continues today as one of the fastest growing, top-tier biomedical research enterprises in the world -- with 46 academic departments, centers, institutes, and programs, and a faculty of more than 3,000 physicians, scientists, and allied health professionals, including members of the National Academy of Medicine and the National Academy of Sciences, and a distinguished two-time winner of the Albert E. Lasker Award in Medical Research. With an operating budget of more than $1.2 billion, the School of Medicine works closely in partnership with the University of Maryland Medical Center and Medical System to provide research-intensive, academic and clinically based care for nearly 2 million patients each year. The School of Medicine has nearly $600 million in extramural funding, with most of its academic departments highly ranked among all medical schools in the nation in research funding. As one of the seven professional schools that make up the University of Maryland, Baltimore campus, the School of Medicine has a total population of nearly 9,000 faculty and staff, including 2,500 student trainees, residents, and fellows. The combined School of Medicine and Medical System (“University of Maryland Medicine”) has an annual budget of over $6 billion and an economic impact of nearly $20 billion on the state and local community. The School of Medicine, which ranks as the 8th highest among public medical schools in research productivity (according to the Association of American Medical Colleges profile) is an innovator in translational medicine, with 606 active patents and 52 start-up companies. In the latest U.S. News & World Report ranking of the Best Medical Schools, published in 2021, the UM School of Medicine is ranked #9 among the 92 public medical schools in the U.S., and in the top 15 percent (#27) of all 192 public and private U.S. medical schools. The School of Medicine works locally, nationally, and globally, with research and treatment facilities in 36 countries around the world. Visit To learn more about Dr. Feldman’s research visit

About The Michael J. Fox Foundation for Parkinson's Research
As the world's largest nonprofit funder of Parkinson's research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson's disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson's patients, business leaders, clinical trial participants, donors and volunteers. In addition to funding $1 billion in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson's research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; increases the flow of participants into Parkinson's disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson's awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world. For more information, visit us on at

Forward-Looking Statements
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, statements regarding the market opportunity for Gain’s product candidates, and the business strategies and development plans of Gain. 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.

Investor & Media Contacts:
Gain Therapeutics Investor Contact:
Daniel Ferry
LifeSci Advisors
+1 (617) 430-7576
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Gain Therapeutics Media Contact:
Joleen Schultz
Joleen Schultz & Associates
+1 760-271-8150
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