8 products found

Gain Therapeutics, Inc. products

Gain - Gaucher Disease

Gaucher disease is the most common lysosomal storage disease and is caused by mutations in GBA, the gene that encodes the beta-glucocerebrosidase (GCase) enzyme. These mutations result in the misfolding and subsequent dysfunction of GCase, which leads to the toxic buildup of fat in a variety of organs and tissues such as the liver, spleen, bones and central nervous system (CNS). While there is currently no cure for these patients, enzyme replacement therapy can be used to address non-CNS symptoms. Gain is developing allosteric regulators that are designed to increase GCase activity and decrease toxic fat accumulation in the CNS of patients with Gaucher disease in order to potentially provide the first treatment approach for the disease’s neuronopathic symptoms.

Gain - Protein Misfolding Technology

Protein misfolding is the root of many diseases, including lysosomal storage disorders and neurodegenerative diseases.

Gain - Krabbe Disease

Krabbe disease is a severe neurodegenerative disorder caused by mutations in GALC, the gene that encodes the galactosylceramidase (GALC) enzyme. These mutations cause GALC misfolding and dysfunction, which leads to the toxic buildup up fats in the central nervous system (CNS) and ultimately leads to the demyelination and death of neurons. There is no cure for this disease and most patients will die before the age of two. Gain is developing allosteric regulators to restore GALC function and potentially limit neuronal cell death, potentially providing the first treatment option to patients who are in desperate need.

Gain - Site-Directed Enzyme Enhancement Therapy (See-Tx) Platform

Gain is utilizing its Site-Directed Enzyme Enhancement Therapy (See-Tx™) platform to guide misfolded enzymes back into their proper shape in order to treat a range of diseases.

Gain - Gangliosidosis Disease

GM1 Gangliosidosis is a hereditary, progressive disease mostly impacting neurons in the brain and spinal cord, caused by mutations in GLB1, the gene that encodes the beta-galactosidase (GLB) enzyme. These mutations result in the misfolding and subsequent dysfunction of GLB, which leads to the toxic substrate accumulation of GM1 ganglioside in organs and tissues. Very limited and investigational symptomatic treatment options include substrate reduction therapy, enzyme replacement therapy, bone marrow transplantation, stem cell transplantation and gene therapy to address non-CNS symptoms. Gain is developing allosteric regulators that are designed to decrease toxic substrate accumulation in organs and tissues of patients with GM1 Gangliosidosis in order to potentially provide the first treatment approach for the disease’s neurological symptoms.