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mRNA Therapies for Dystrophic Epidermolysis Bullosa (DEB)

Description

Dystrophic Epidermolysis Bullosa (DEB) is a disease caused by mutations in the COL7A1 gene, which encodes collagen 7. Many DEB patients have extremely delicate skin, and in the most severe cases, live with painful blisters and open wounds over large areas of their bodies.

Our scientists developed a method for treating dystrophic epidermolysis bullosa by using mRNA to express collagen 7 directly in a patient’s skin.

The mRNA Therapies for Dystrophic Epidermolysis Bullosa (DEB) technology is protected by AU Patent Number 2015210769 (with additional patents pending in the U.S. and in other countries). Of note, the granted patent includes claims that are not limited by specific mRNA sequence, chemistry or formulation.


Example Applications

  • Directly express collagen 7 in DEB patient skin
  • Develop a single product to treat a complex population with hundreds of disease-causing mutations
  • Treat large areas using a microneedle array
  • Dose infrequently – collagen 7 has a long in vivo half-life (>30 days)
  • Combine with Factor’s ToRNAdo™ Nucleic-Acid Delivery System for high efficiency in vivo delivery – proven delivery to human skin in vivo

Representative Claim

AU Pat. No. 2015210769
An in vivo method for treating a dystrophic epidermolysis bullosa patient comprising

delivering a synthetic RNA encoding collagen type VII to the patient's keratinocytes by injection to the epidermis, thereby resulting in the amelioration of one or more of the dystrophic epidermolysis bullosa patient's symptoms.