mTORC1 Inhibitors for Fibrosis

Fibrosis is a disease of metabolism of fibroblasts, or fibrometabolism, which causes the thickening and scarring of connective tissue. Fibrosis can occur in many tissues in the body such as the lung, liver and heart, and may ultimately cause disease by altering the architecture and function of the underlying organ or tissue.

Research has shown hyperactive mTOR pathway activity in specific cells called myofibroblasts that are found in fibrotic foci in the lung and kidney. Genetic or pharmacological inhibition of mTOR can prevent activation of myofibroblasts and secretion of extracellular matrix in experimental models and prevent fibrosis. Our fibrosis program targets the selective inhibition of mTORC1 signaling through the modulation of glucose metabolism and has demonstrated efficacy in a validated model of pulmonary fibrosis.

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