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Therapeutic Pipeline: Innovating Treatments for Unmet Needs

Leading the Discovery of Allosteric Binding Sites to Create New Medicines
PROCESS FLOW
Gain Therapeutics Inc.
ASSET
Indication
Target
Discovery
Research
Preclinical
Phase 1
Neurodegeneration
Lysosomal Storage Disorders
Metabolic Disorders
Oncology
Lead Program
Lead Program

Gain Therapeutics’ lead drug candidate, GT-02287, is an allosteric regulator of GCase and is in development for the treatment of PD, with GBA1-PD as the lead indication. The orally administered, brain-penetrant small molecule restores the function of GCase. In preclinical models of PD, GT-02287 restored GCase enzymatic function, reduced cellular stress in the endoplasmic reticulum, improved lysosomal function and mitochondrial function, reduced toxic glycosphingolipids, 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.

Gain is conducting a Phase 1b clinical trial of GT-02287 in people with GBA1 and idiopathic Parkinson’s disease. Interim data from the Phase 1b clinical trial is expected mid-2025.

More information about Gain’s Phase 1b clinical trial can be found here

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