UCHL1 Program

UCHL1 (Ubiquitin C-terminal hydrolase 1); a DUB disease target for fibrotic disease

Lead optimization program

Therapeutic Rationale

UCHL1 is novel functional marker for liver myofibroblasts and a therapeutic target in chronic liver disease (Wilson et al., 2015). It is also strongly expressed in clinical samples of idiopathic pulmonary fibrosis (IPF) and Chronic kidney disease (CKD). UCHL1 inhibition has been shown to reduce liver fibrosis in in vivo models and reduce fibrogenesis in several liver fibrosis models. Mission has identified potent and selective inhibitors of UCHL1.

About Fibrosis

Fibrosis can occur in many tissues of the body as a result of inflammation or damage. This leads to build up of collagen, which can produce scarring of vital organs such as the lung, liver, skin, eye, heart and kidney, resulting in irreversible damage and eventual organ failure. Fibrosis represents a large unmet clinical need as it is estimated to be prevalent in 45% of all diseases.

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