LU 2015-101

Inventors

Ann Harris*

Lindsay Stolzenburg

Short Description

A microRNA drug candidate that treats fibrotic diseases through the repression of TGF-β receptor expression.

Abstract

Northwestern researchers have identified an innovative therapeutic strategy to treat acute and chronic fibrotic diseases. Progression of fibrosis involves repeated cycles of inflammation, epithelial injury and repair, which in turn, causes scarring and tissue malfunction. Chronic fibrotic diseases, such as cystic fibrosis, often result in irreversible damage to the fibrotic organ and the need for transplantation. Currently, there is no available treatment to halt the progression of fibrosis. Northwestern researchers have identified a therapeutic candidate that capitalizes on the role of transforming growth factor beta (TGF-β). TGF-β is a cytokine that is normally released in response to injury to stimulate wound repair. However, its overproduction is associated with tissue scarring in the fibrosis process. This therapeutic candidate is a novel microRNA which can repress the expression of two canonical TGF-β receptors, TGFBR1 and TGFBR2, and substantially decrease downstream TGF-β signaling. Unlike current therapeutics which only offer temporary anti-inflammatory relief, this compound would be the first therapeutic to target a specific upstream modulator involved in the progression of fibrosis.

Applications

• Treatment of fibro-proliferative diseases including:
• Pulmonary fibrosis
• Liver cirrhosis
• Myocardial fibrosis
• Systematic sclerosis
• Renal fibrosis
• Autoimmune diseases
• Research tool to reduce TGF-β signaling in vitro or in vivo

Advantages

• Versatile: Applicable to all types of TGF-β induced fibrosis
• Cost-effective: Compared to the cost of antibody drug development

Publications

Stolzenburg L, Wachtel S, Dang H and Harris A (2016) MIR-1343 attenuates pathways of fibrosis by targeting the TGF-β receptors. Biochemical Journal. 473 (3): 245-56.

IP Status

Issued US Patent No. 9,856,481