Gain Therapeutics Presents Compelling Preclinical Data For Parkinson’s Disease Drug Candidate

Gain Therapeutics, Inc. (Nasdaq: GANX) has announced preclinical data demonstrating that its lead drug candidate GT-02287 significantly decreased Parkinson’s disease (PD)-associated pathology and improved motor dysfunction in two different preclinical models of Parkinson’s disease.

Matthias Alder, Chief Executive Officer of Gain Therapeutics, said, “The data…demonstrate that our lead drug candidate GT-02287 can restore GCase protein function, protect against the neuropathological features of Parkinson’s disease, and reduce neurodegeneration measured by the emerging biomarker NfL. We look forward to initiating the Phase 1 clinical study with GT-02287 and advancing the development of this potentially best-in-class treatment for GBA1 Parkinson’s disease.”


Gain’s studies found that GT-02287 significantly reduced plasma Neurofilament Light Chain (NfL) levels, an emerging biomarker for neurodegeneration, in a mouse model of GBA1-PD.

The study showed that orally-administered GT-02287 restored enzymatic function and significantly reduced the pathological hallmarks and motor dysfunction associated with Parkinson’s disease.

GT-02287 also decreased plasma NfL, which is an emerging and important neurodegeneration biomarker that can be measured in blood.

The company’s findings are being presented in two posters, including one that was accepted as a late breaking abstract, at the International Congress of Parkinson’s Disease and Movement Disorders® being held in Copenhagen, Denmark from August 27-31, 2023.

About Gain Therapeutics

Gain Therapeutics, Inc. is a biotechnology company leading the discovery and development of next generation allosteric therapies. Gain’s lead drug candidate GT-02287, in development for the treatment of GBA1 Parkinson’s disease, has completed IND-enabling GLP toxicology studies. Clinical evaluation of GT-02287 is expected to begin this year.

Leveraging AI-supported structural biology, proprietary algorithms and supercomputer-powered physics-based models, the company’s SEE-Tx® discovery platform can identify novel allosteric binding sites on disease-implicated proteins, pinpointing pockets that cannot be found or drugged with current technologies. 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.

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Gain Therapeutics Presents Compelling Preclinical Data For Parkinson’s Disease Drug Candidate

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