Gain Therapeutics

Gain Therapeutics to Present Phase 1 Data for Novel Parkinson's Drug GT-02287 at MJFF Conference

3 months ago

• Gain Therapeutics will present Phase 1 results for GT-02287, their brain-penetrant small molecule drug candidate for Parkinson's disease, at the Michael J. Fox Foundation's 16th Annual Conference. • GT-02287 demonstrated favorable safety, target engagement, and therapeutic exposure in healthy volunteers, showing promise as a potential disease-modifying treatment for both GBA1-mutation and idiopathic Parkinson's disease. • The presentation will take place during the "Progress in the Therapeutics Pipeline" session on October 17, 2024, in New York City, highlighting recent preclinical and clinical developments.

Gain Therapeutics' GT-02287 Enters Phase 1b Trial for Parkinson's Disease

5 months ago

• Gain Therapeutics has initiated a Phase 1b clinical trial in Australia for GT-02287, targeting both idiopathic and GBA1-linked Parkinson's disease. • The trial aims to evaluate the safety, tolerability, and biomarker changes over three months of GT-02287 administration in Parkinson's patients. • GT-02287 previously demonstrated a favorable safety profile and significant GCase enzyme engagement in a Phase 1 study with healthy volunteers. • Interim results from the Phase 1b trial are expected by mid-2025, potentially paving the way for Phase 2 trials in the U.S. and EU.

CAP-003 Gene Therapy Shows Promise for Parkinson's Disease with GBA Mutations

7 months ago

• Capsida Biotherapeutics' CAP-003 gene therapy demonstrated over 200-fold greater RNA expression in nonhuman primates compared to AAV9, suggesting enhanced efficacy. • CAP-003 significantly increased GCase protein and enzyme activity in the brain, exceeding the efficacy threshold needed for normalizing enzyme activity in humans. • The gene therapy reduced glucosylsphingosine levels in plasma, indicating target engagement and improved lysosomal function, crucial for slowing disease progression. • Capsida plans to initiate clinical trials for CAP-003 in the first half of 2025, marking a significant step toward a potential disease-modifying treatment.

Gain Therapeutics' GT-02287 Shows Promise in Phase 1 Trial for Parkinson's Disease

9 months ago

• Gain Therapeutics announced positive topline results from its Phase 1 clinical trial of GT-02287, a novel small molecule therapy targeting GCase for Parkinson's disease. • The study demonstrated that GT-02287 was safe and generally well-tolerated in healthy volunteers, with evidence of central nervous system (CNS) exposure and target engagement. • A Phase 1b trial in Parkinson's disease patients is planned for Q4 2024, with data expected by mid-2025 to assess safety, tolerability, and proof of mechanism. • GT-02287 is an orally administered, brain-penetrant molecule designed to restore GCase function, reduce α-synuclein aggregation, and improve motor and cognitive functions.