BETHESDA, Md. - Gain Therapeutics, Inc. (NASDAQ:GANX), a biotechnology company engaged in developing allosteric small molecule therapies, has shared preclinical data suggesting its GCase regulator GT-02287 could offer neuroprotection and restore motor function in Parkinson’s disease models, even with delayed administration. The findings were presented as a late-breaker abstract at the 20th WORLDSymposium™ in San Diego this week.
The study indicated that GT-02287, when administered to a mouse model of Parkinson’s disease after the onset of disease symptoms, not only improved motor functions but also reduced the levels of Neurofilament Light Chain (NfL), a biomarker associated with neurodegeneration, to those of the control group. These results suggest the drug candidate has a progressive effect on reversing motor deficits with continued treatment.
Matthias Alder, CEO of Gain Therapeutics, expressed optimism about the potential of GT-02287 to slow or halt the progression of Parkinson’s disease, for which only symptomatic treatments are currently available. The company is conducting a Phase 1 clinical trial to assess the safety, tolerability, and pharmacokinetics of GT-02287 in healthy adults and plans to extend the trial to include patients with Parkinson’s disease in the third quarter of this year.
GT-02287 is designed to cross the blood-brain barrier and modulate the function of the lysosomal protein glucocerebrosidase (GCase). In patients with GBA1 Parkinson’s disease, a mutation in the GBA1 gene leads to a dysfunctional enzyme, contributing to the accumulation of alpha-synuclein and the degeneration of dopamine-producing neurons. Gain Therapeutics’ drug candidate aims to correct this by restoring GCase function, potentially modifying the disease's course.
The program has received funding support from The Michael J. Fox Foundation for Parkinson’s Research, The Silverstein Foundation for Parkinson’s with GBA, and InnoSuisse.
This press release contains forward-looking statements about the potential benefits and clinical development of GT-02287. The actual results could differ materially from those projected in the forward-looking statements due to a variety of risks and uncertainties. The information reported is based on a press release statement from Gain Therapeutics.
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