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Gain Therapeutics Announces 2024 R&D Update on Parkinson’s Disease with KOL & Analyst Insight

Event, moderated by Hartaj Singh and Eka Gigauri of Oppenheimer & Co., will be held on Thursday, February 22, 2024 at 11:00 am EST

BETHESDA, Md., Feb. 14, 2024 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc., (Nasdaq: GANX), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today announced it is holding a 2024 Research & Development update on recent in vivo data for GT-02287 along with valuable insights from Key Opinion Leaders.

Event Details

“Beyond Symptomatic Treatment of Parkinson’s Disease: Gain Therapeutics R&D Update with KOL & Analyst Insights 2024”

Date: February 22nd, 2024

Time: 11:00 am - 12:00 pm EST

Register for the Event HERE.

Send any questions you’d like answered in our Q&A to This email address is being protected from spambots. You need JavaScript enabled to view it..

Discussion Topics

GBA-associated Parkinson’s Disease & other Synucleinopathies: current unmet medical needs and needed therapies.

From biology to classification: understanding Parkinson’s Disease.

Discussion of recent in vivo data for GT-02287 as a best-in-class small molecule, for the treatment of GBA-Parkinson’s Disease and other neurodegenerative diseases.


Gain Therapeutics

Matthias Alder - Chief Executive Officer

Joanne Taylor – Senior Vice President, Research

Key Opinion Leaders

Professor Roy Alcalay

  • Prof. Alcalay is Chief of the Movement Disorders Division at Tel Aviv Sourasky Medical Center in Tel Aviv, Israel. He also holds an associate professorship in clinical neurology at Columbia University. His research focuses on biomarkers and genetics in Parkinson’s Disease. He is a member of The Michael J. Fox Foundation-funded Parkinson’s Progression Markers Initiative (PPMI) executive steering committee, and the PI of the Parkinson’s Foundation-funded PD GENEration study.

Professor Tiago Fleming Outeiro

  • Prof. Outeiro is Professor and Director of the Department of Experimental Neurodegeneration at the University Medical Center Göttingen in Germany. He is also Professor at Newcastle University. His research interests are focused on the understanding of the molecular mechanisms of neurodegenerative diseases such as Parkinson’s, Huntington’s, and Alzheimer’s disease. He is Chair of the Task Force on the Biological Definition of Parkinson’s Disease of Internation Parkinson and Movement Disorder Society.

About GT-02287

Gain Therapeutics’ lead drug candidate, GT-02287, currently being evaluated in a Phase 1 clinical trial, has the potential to be a disease-modifying treatment of GBA1 Parkinson’s disease (GBA1-PD) and other neurodegenerative diseases. The orally administered small molecule that crosses the blood-brain barrier is an allosteric protein modulator that restores the function of the lysosomal protein glucocerebrosidase (GCase). In GBA1-PD patients, this enzyme 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 and reduced aggregated α-synuclein, neuroinflammation and neuronal death, and caused increased striatal dopamine levels and improved motor function. Additionally, GT-02287 significantly reduced plasma neurofilament light chain (NfL) levels, an emerging biomarker of neurodegeneration.

The program has been awarded funding support from The Michael J. Fox Foundation for Parkinson’s Research (MJFF), The Silverstein Foundation for Parkinson’s with GBA, and InnoSuisse.

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-producing neuronal cells. Patients with GBA1-PD tend to have earlier onset and faster symptom progression than 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™ 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.

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 trial for GT-02287 and the treatment of Parkinson’s patients in that clinical trial; and the potential therapeutic and clinical benefits of the Company’s product candidates including GT-02287. 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.

Investor Contact:

(516) 222-2560 
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Media Contacts:

Russo Partners
Nic Johnson and Elio Ambrosio
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(212) 845-4242