UPDATE - Gain Therapeutics’ GT-02287 Completely Restores Motor Function in Mouse Models of Parkinson’s Disease - Seite 2

Further details of the study, including protocol and specific results can be found in the poster, which was presented today and can be accessed here.

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.