PIPELINE
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Alpha-1 antitrypsin (AAT) deficiency is an inherited disorder that can cause disease in the lung and liver. Symptoms include inability to breathe, recurring respiratory infections and fatigue as well as liver cirrhosis, leaving patients at risk for development hepatocellular carcinoma. AAT deficiency is caused by mutations in SERPINA1, the gene that encodes the AAT protein, a serine protease inhibitor or serpin. The AAT protein is made in the liver and plays a crucial role in protecting lung function.
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Solid tumors are abnormal masses of tissue that occur in bones, muscles or other organs. Solid tumors may be cancerous or non-cancerous. Some of the most common solid tumor types occur in the breast, colon, bladder, prostate, and lung.
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 1 clinical trial of GT-02287 in healthy volunteers with expected completion in the first half of 2024 and plans to initiate a patient cohort in that clinical trial in the third quarter of 2024.
More information about clinical trials can be found here