This technology is a customizable exosome system for the targeted delivery of therapeutic RNAs to the liver to prevent pathogenic fibrosis.
Liver fibrosis and associated diseases account for more than two million deaths per year globally. Despite this massive healthcare burden, there are currently no FDA-approved therapeutics for liver fibrosis. Furthermore, most drugs in development address only inflammation, and fail to specifically target fibrotic buildup within the liver. Therefore, there is a pressing need for a system that can precisely deliver anti-fibrotic treatments to the liver to achieve meaningful therapeutic benefit.
This technology uses an engineered exosome platform to target and safely deliver an anti-microRNA to the liver. The cargo, LNA-anti-miR-132, inhibits the activity of miR-132, which has been associated with liver fibrosis and disease. Administration of this drug decreases the level of collagen deposition, decreases the levels of pro-fibrotic gene expression, and promotes the expression of anti-fibrotic proteins, while limiting off-target effects.
This technology has been validated with mouse models of liver fibrosis.
IR CU21066
Licensing Contact: Joan Martinez