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:

Fatemeh Momen-Heravi, PhD

Patent Information:

Patent Status

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