mRNA Delivery to Skin

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In addition to being the largest and most accessible organ of the body, the skin contains large numbers of highly active cells that have a high capacity for protein synthesis. These characteristics make the skin an ideal platform for expressing therapeutic proteins, both locally, for the treatment of dermatological conditions, and systemically, for the treatment of a wide range of diseases and conditions.

Our scientists developed a method for expressing therapeutic proteins, including circulating proteins, by administering ultra-low doses of mRNA to the skin.

mRNA Delivery to Skin is protected by U.S. Patent Number 11,241,505 (with additional patents pending in the U.S. and in other countries). Of note, the granted patent includes claims that are not limited by disease indication, cell type, target sequence, type of gene-editing protein, method of transfection, mRNA sequence or chemistry, or formulation.

Example Applications

  • Express proteins locally for the treatment of dermatologic conditions (e.g., elastin for the rare genetic disease cutis laxa and aesthetic applications)
  • Express proteins systemically for the treatment of a wide range of diseases and conditions (e.g., BMP7 for diabetic nephropathy)
  • Deliver the therapy directly to the patient’s skin – avoid ex vivo cell manipulation
  • Combine with Factor’s Chromatin Context-Sensitive Gene-Editing Endonuclease for high-specificity in vivo gene editing
  • Combine with Factor’s ToRNAdo™ Nucleic-Acid Delivery System for high efficiency in vivo delivery – proven delivery to human skin in vivo

Representative Data

Figure 1. Rat skin containing cells expressing a reporter protein following mRNA delivery.

Representative Claim

U.S. Pat. No. 11,241,505

A method for gene-editing a cell in the skin of a human subject, comprising inducing a single-strand or double-strand break in the DNA of the cell, comprising:

administering intradermally to the human subject a composition comprising an effective dose of about 300 ng of an RNA encoding a protein of interest to result in the cell expressing the protein of interest,

wherein the protein of interest is a gene-editing protein, and

wherein the effective dose of about 300 ng of the RNA encoding the protein of interest provides greater expression of the protein of interest than a dose of 1200 ng of RNA encoding the protein of interest during the second to fifth day after administering the RNA.