Targeted delivery of anti-microRNA for the treatment of liver fibrosis
This technology is a customizable exosome system for the targeted delivery of therapeutic RNAs to the liver to prevent pathogenic fibrosis.
Unmet Need: Improved liver-specific delivery of anti-fibrotic therapeutics
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.
The Technology: Liver-targeting exosomes for the delivery of fibrosis inhibitors
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.
Applications:
- Induction or inhibition of liver fibrosis
- Treatment of cancer
- Management of viral infections
- Delivery of nucleic acid-based therapeutics
- Investigation of the role of micro-RNA function
Advantages:
- Increased stability of nucleic acid cargo
- Organ-specificity reduces systemic toxicity and off-target effects
- Can be modified to target particular tissues
- Encapsulation minimizes aberrant immune response
- Ability to cross the plasma membrane
Lead Inventor:
Patent Information:
Related Publications:
Tech Ventures Reference:
IR CU21066
Licensing Contact: Joan Martinez