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Gain Therapeutics Presents Data at the AD/PD™ 2024 Conference Demonstrating the Mechanism of Action of GT-02287, its Clinical Stage GCase Regulator for the Treatment of Parkinson’s Disease

Data support the ability of GT-02287 to prevent several steps in the disease cascade resulting from GCase misfolding and dysfunction

BETHESDA, Md., March 05, 2024 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today announces the presentation of a poster at the International Conference on Alzheimer's and Parkinson's Diseases and Related Neurological Disorders (AD/PD™ 2024) that addresses the mechanism of action of the Company’s lead compound, GT-02287. AD/PD™ 2024 is being held March 5th – 9th, 2024 in Lisbon, Portugal.

The poster, titled “GT-02287, a Clinical Stage Glucocerebrosidase Regulator for the Treatment of PD, Eases ER Stress and Enhances Lysosomal Enzyme Activity,” which will be available online March 5th and presented on-site on the 8th and 9th by Dr. Natalia Perez-Carmona, demonstrates how GT-02287, through its interaction with glucocerebrosidase (GCase) in the endoplasmic reticulum (ER), aids correct GCase folding, preventing ER retention, ER stress, and ER-associated degradation of mutated GCase enzyme. GCase is consequently able to travel to the lysosome, resulting in enhanced lysosomal activity and efficient processing of the GCase substrate glucosylceramide. Increase in GCase substrate in the lysosome was previously shown to be associated with accumulation of aggregated alpha-synuclein, a pathological hallmark of Parkinson’s disease and related disorders. The study was done in collaboration with scientists in the group of Professor Maurizio Molinari at the Università dela Svizzera italiana-affiliated Institute for Research in Biomedicine in Bellinzona, Switzerland and was conducted under the support of a Eurostars-2 joint program.

“These data further confirm our understanding of the mechanism of action of GT-02287, how it impacts cellular health by preventing the downstream consequences of GCase misfolding, including cellular stress and lysosomal dysfunction” commented Dr. Natalia Perez-Carmona, Senior Director of Biology at Gain Therapeutics and presenting author. “The AD/PD™ conference is an important opportunity for us to continue to discuss our differentiated approach to addressing GBA1 Parkinson’s disease through GCase modulation among this key audience.”

A PDF of the poster presented at the AD/PDTM 2024 conference is available on the Science & Technology section of the Company’s website at

The AD/PD™ Conference is an annual event attracting leading medical and scientific professionals from around the world, focusing on the advances of science and therapy of Alzheimer’s and Parkinson’s Diseases and related neurological disorders.

About GT-02287

Gain Therapeutics’ lead drug candidate, GT-02287, is in clinical development for the treatment of GBA1 Parkinson’s disease (GBA1-PD). The orally administered, brain-penetrant small molecule is an allosteric protein modulator that restores the function of the lysosomal protein enzyme glucocerebrosidase (GCase) which becomes misfolded and impaired due to a GBA1 gene mutation, the most common genetic abnormality associated with PD. In preclinical models of PD, GT-02287 restored GCase enzymatic function, reduced aggregated α-synuclein, neuroinflammation and neuronal death, increased dopamine levels and improved motor function. Additionally, GT-02287 significantly reduced plasma neurofilament light chain (NfL) levels, an emerging biomarker for neurodegeneration.

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 – Swiss Innovation Agency.

About GBA1 Parkinson’s Disease

GBA1 Parkinson’s disease is caused by mutations in the GBA1 gene, found in up to 15% of patients with Parkinson’s disease and making it the primary genetic risk factor. The mutation causes dysfunctional misfolding of the lysosomal enzyme glucocerebrosidase (GCase), reducing its activity in the brain and leading to the subsequent accumulation of α-synuclein and degeneration of dopamine-containing nerve cells. Patients with GBA1-PD tend to have earlier onset and faster symptom progression than those with sporadic PD, a progressive neurodegenerative disease characterized by a motor syndrome consisting of bradykinesia (slowness of movement), rigidity, resting tremors, and postural instability. With current therapies treating only the symptoms of Parkinson’s disease without affecting the underlying progression of the disease, there is an unmet need to develop novel disease-modifying therapies such as GT-02287 that have the potential to slow or stop disease progression and help improve outcomes in this patient population.

About Gain Therapeutics, Inc.

Gain Therapeutics, Inc. is a clinical-stage biotechnology company leading the discovery and development of next generation allosteric therapies. Gain’s lead drug candidate GT-02287 for the treatment of GBA1 Parkinson’s disease, is currently being evaluated in a Phase 1 clinical trial.

Leveraging AI-supported structural biology, proprietary algorithms and supercomputer-powered physics-based models, the company’s Magellan™ drug discovery platform can identify novel allosteric binding sites on disease-implicated proteins, pinpointing pockets that cannot be found or drugged with current technologies. Magellan™ is the next generation of Gain’s original SEE-Tx® (Site-Directed Enzyme Enhancement Therapy) platform, which was enhanced and expanded with new AI and machine-learning tools and virtual screening capabilities to access the emerging on-demand compound libraries covering vast chemical spaces of over 50 billion compounds.

Gain’s unique approach enables the discovery of novel, allosteric small molecule modulators that can restore or disrupt protein function. Deploying its highly advanced platform, Gain is accelerating drug discovery and unlocking novel disease-modifying treatments for untreatable or difficult-to-treat disorders including neurodegenerative diseases, rare genetic disorders and oncology. For more information, please visit and follow us on LinkedIn.

About Institute For Research in Biomedicine (IRB, Bellinzona)

Founded in 2000 in Bellinzona, Switzerland, the Institute for Research in Biomedicine (IRB) hosts 13 research groups studying infectious, inflammatory, rare diseases and tumors in order to identify new therapeutic strategies. As a result of its activities, the IRB has gained considerable international recognition in the fields of immunology, cell and structural biology and DNA repair. Molinari’s laboratory investigates the molecular mechanisms regulating chaperone-assisted protein folding and the quality control processes determining whether a polypeptide should be transported at the intra- or extra-cellular site of activity, or if it should be selected for degradation. Emphasis is given on establishing causes of diseases resulting from misfolding of mutant gene products such as α1-antitrypsin deficiency and lysosomal storage disorders and to explore novel therapeutic approaches to alleviate disease phenotypes. IRB offers training opportunities for young researchers at all levels. This is made possible through collaborations with Swiss and foreign universities. Since its opening, IRB has trained more than 120 PhD students who have gone on to careers in academia or industry both in Switzerland and abroad. In 2021, IRB and IOR (Institute of Oncology Research) formed the Bios+ (Bellinzona Institutes of Science) association, with the mission to create new synergies and to promote and coordinate the scientific research and teaching activities of the two institutes. The vision of Bios+ is to promote the growth of a biomedical research center in Italian-speaking Switzerland. For more information, please visit

Cautionary Note Regarding Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. All statements in this press release other than statements of historical facts are “forward-looking statements”. In some cases, you can identify these statements by forward-looking words such as "may," "might," "will," "should," "expect," "plan," "anticipate," "believe," "estimate," "predict," "goal, " "intend," "seek, " "potential" or "continue," the negative of these terms and variations of these words or similar expressions that are intended to identify forward-looking statements, although not all forward-looking statements contain these words. Forward-looking statements in this press release include, but are not limited to, statements regarding: the development of the Company’s current or future product candidates including GT-02287; expectations regarding the timing of results from a Phase 1 clinical study for GT-02287; and the potential therapeutic and clinical benefits of the Company’s product candidates. These forward-looking statements are based on the Company’s expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties that could cause the Company’s preclinical and future clinical development programs, future results or performance to differ materially from those expressed or implied by the forward-looking statements. These statements are not historical facts but instead represent the Company's belief regarding future results, many of which, by their nature, are inherently uncertain and outside the Company's control. Many factors may cause differences between current expectations and actual results, including the impacts of the post-COVID-19 environment and other global and macroeconomic conditions on the Company’s business; clinical trials and financial position; unexpected safety or efficacy data observed during preclinical studies or clinical trials, clinical trial site activation or enrollment rates that are lower than expected; changes in expected or existing competition; changes in the regulatory environment; the uncertainties and timing of the regulatory approval process; and unexpected litigation or other disputes. Other factors that may cause the Company’s actual results to differ from those expressed or implied in the forward-looking statements in this press release are identified in the section titled “Risk Factors,” in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 23, 2023 and its other documents subsequently filed with or furnished to the Securities and Exchange Commission from time to time. All forward-looking statements contained in this press release speak only as of the date on which they were made. The Company undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

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