Gain Therapeutics to Join H.C. Wainwright Neuro Perspectives Conference

Gain Therapeutics, Inc., a biotechnology company at the clinical-stage, is making strides in the discovery and development of innovative allosteric small molecule therapies. On June 27, 2024, the company's Chief Financial Officer, Gene Mack, will be featured in a fireside chat and will conduct virtual one-on-one meetings at H.C. Wainwright’s 5th Annual Neuro Perspectives Virtual Conference. The presentation will be accessible on-demand and an archived replay will be available on the company’s website.

The company’s lead drug candidate, GT-02287, is in clinical development for the treatment of GBA1 Parkinson’s disease (GBA1-PD). GT-02287 is an orally administered, brain-penetrant small molecule that acts as an allosteric modulator, helping to restore the function of the lysosomal protein enzyme glucocerebrosidase (GCase). This enzyme often becomes misfolded and impaired due to a mutation in the GBA1 gene, which is the most common genetic abnormality linked to Parkinson’s disease.

In preclinical models of Parkinson’s disease, GT-02287 has shown promising results. It restored GCase enzymatic function, decreased aggregated α-synuclein and neuroinflammation, reduced neuronal death, and enhanced dopamine levels. Additionally, it improved motor functions and cognitive performance. GT-02287 also significantly lowered plasma neurofilament light chain (NfL) levels, which is an emerging biomarker for neurodegeneration.

The development of GT-02287 has garnered support from various esteemed organizations. The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and The Silverstein Foundation for Parkinson’s with GBA have provided funding. Additionally, the Eurostars-2 joint program, co-funded by the European Union Horizon 2020 research and Innosuisse – Swiss Innovation Agency, has also supported this project.

Gain Therapeutics, Inc. is a pioneering company in the field of biotechnology, specializing in the discovery and development of the next generation of allosteric therapies. The company's innovative drug candidate GT-02287 is currently undergoing a Phase 1 clinical trial for the treatment of GBA1 Parkinson’s disease.

The company's drug discovery is powered by the Magellan™ platform, which leverages AI-supported structural biology, proprietary algorithms, and supercomputer-powered physics-based models. This advanced platform enables the identification of novel allosteric binding sites on disease-implicated proteins, including pockets that are not accessible with current technologies. The AI and machine learning tools, along with virtual screening capabilities, enable the exploration of a vast chemical space of over five trillion compounds to identify and select suitable small molecule hits for experimental validation.

Gain’s unique and cutting-edge approach allows for the discovery of novel allosteric small molecule modulators that can restore or disrupt protein function. This innovation accelerates the drug discovery process and opens new avenues for developing treatments for untreatable or difficult-to-treat disorders, including neurodegenerative diseases, rare genetic disorders, and oncology.

Gain Therapeutics, Inc. continues to lead in this innovative field, leveraging the power of advanced computational and experimental techniques to discover and develop novel therapeutic solutions that address significant unmet medical needs.