Gain Therapeutics to Present at the Cambridge Healthtech Institute’s 2nd Annual Neurodegeneration Targets Conference

BETHESDA, Md., Sept. 20, 2023 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc., (Nasdaq: GANX) (“Gain”, or the “Company”), a biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today announced that Matthias Alder, Chief Executive Officer of Gain Therapeutics, will deliver a featured presentation at the Cambridge Healthtech Institute’s 2nd Annual Neurodegeneration Targets Conference: Drug Discovery for Progressive Central Nervous System Disorders. The event is taking place at the Sheraton Boston in Boston, Massachusetts, between September 26-27, 2023.

Presentation Details
Segment: Targeting Glucocerobrosidase (GCase) for Parkinson’s Disease
Featured Presentation: Allosteric Activators of GCase
Date & Time: Tuesday, September 26, 2023 at 8:35 a.m. ET
Register for the Event here

About GT-02287

Gain Therapeutics’ lead drug candidate, GT-02287, is in development for the treatment of GBA1 Parkinson’s disease (GBA1-PD). The orally available, brain-penetrant small molecule is an allosteric protein modulator that restores the function of the lysosomal protein enzyme glucocerebrosidase (GCase) which 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, reduced aggregated α-synuclein, neuroinflammation and neuronal death, increased dopamine levels and improved motor function. Additionally, GT-02287 significantly reduced plasma neurofilament light chain (NfL) levels, an emerging biomarker for neurodegeneration.

The program has been awarded funding support from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and The Silverstein Foundation for Parkinson’s with GBA. A Phase 1 trial of GT-02287 is scheduled to begin in the near term.

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 subsequent neurodegeneration of dopamine 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.