Gain Therapeutics Announces Positive Preclinical Data for GT-02287

Gain Therapeutics, Inc. recently shared encouraging preclinical data regarding its product candidate GT-02287. On October 7, 2024, the company unveiled details of their findings in a press release highlighting the potential of GT-02287. The information disclosed sheds light on the therapeutic benefits observed in preclinical animal models of GBA-1 and idiopathic Parkinson’s disease.

In the animal models representing GBA-1 and Parkinson’s disease, administration of GT-02287 demonstrated persistent rescue of motor and cognitive function. Notably, even after discontinuation of GT-02287, performance levels remained largely unaffected for over a week compared to continuous treatment. The presentations also emphasized GT-02287’s positive outcomes on mitochondrial function, neuroprotection, and reduction in Tau accumulation across various cell lines.

Furthermore, the study evaluated GT-02287’s impact post-clinical phenotype establishment in both GBA-1 and Parkinson’s disease models. Results indicated that GT-02287 not only rescued deficits in neuromuscular function and motor coordination but also prevented the onset of cognitive impairments and deficits in daily activities. Withdrawal of GT-02287 for more than a week did not significantly impact performance in the conducted tests.

In additional tests simulating PD-related dysfunctions, GT-02287 showcased a positive effect on mitochondrial reactive oxygen species, lysosomal pathology, α-synuclein aggregation, and neuroprotection. The compound exhibited the ability to reduce levels of relevant neurodegeneration markers and restore motor function to control levels, indicating promising therapeutic potential.

The data also explored the impact of GT-02287 on lysosomal Tau accumulation in human fibroblasts, revealing a reduction in Tau accumulation in cells carrying GBA mutations.

The company presented these findings as part of its commitment to driving innovation in the field of therapeutics for neurodegenerative diseases. Gain Therapeutics aims to address unmet needs in the treatment landscape, offering new avenues for advancing patient care and outcomes.

Investors and industry stakeholders are keenly observing Gain Therapeutics as it continues to progress with its product pipeline and therapeutic innovations in the realm of neurodegenerative diseases. The company’s ongoing research and development efforts underscore a dedication to pioneering solutions that could potentially transform patient care and clinical outcomes.

This article was generated by an automated content engine and was reviewed by a human editor prior to publication. For additional information, read Gain Therapeutics’s 8K filing here.

About Gain Therapeutics

Gain Therapeutics, Inc, a biotechnology company, develops novel small molecule therapeutics to treat diseases across various therapeutic areas. Its drug discovery platform Magellan discovers novel allosteric binding sites in a disease; identifies proprietary small molecules that bind these sites to modulate protein function; and treats the underlying cause of the disease.