Researchers use our
technologies in their quest to
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HIV, and Parkinson’s

ToRNAdo™ Nucleic-Acid Delivery System

Description

Delivery systems can be used to enhance the uptake of nucleic acids by cells. Conventional delivery systems often suffer from endosomal entrapment and toxicity, which can limit their therapeutic use.

Our scientists developed a novel chemical substance that is exceptionally effective at delivering nucleic acids, including mRNA, to cells both ex vivo and in vivo.

The ToRNAdo™ Nucleic-Acid Delivery System is protected by three U.S. Patents (with additional patents pending in the U.S. and in other countries). Of note, certain granted patents include claims that are not limited by disease indication, cell type, route of administration, or type of nucleic acid.


Example Applications

  • Use fusogenic lipid/nucleic-acid particles made with ToRNAdo™ to avoid endocytosis pathways that require “endosomal escape”
  • Generate non-toxic formulated nucleic-acid products (ToRNAdo™ is made using omega-6 unsaturated tails derived from sunflower seed oil)
  • Achieve ultra-high-efficiency transfection in up to 100% serum
  • Protect cargo from nuclease attack
  • Efficiently delivery of mRNA, siRNA, and plasmid to a variety of cell types
  • Deliver nucleic acids, including mRNA, In vivo – proven delivery to brain, eye, skin, and lung3
  • Use Factor’s high-yield synthesis protocol to streamline nucleic-acid therapy manufacturing
  • Combine with Factor’s mRNA Cell Reprogramming technology to generate footprint-free pluripotent stem cells
  • Combine with Factor’s Chromatin Context-Sensitive Gene-Editing Endonuclease for high-specificity in vivo gene editing

Figure 1. ToRNAdo™ delivery of GFP mRNA to human epidermal keratinocytes in vitro.


Figure 2. ToRNAdo™ delivery of RFP mRNA to human skin in vivo.

Representative Claim

U.S. Pat. No. 10,501,404

A compound of Formula (I)

,

wherein n is 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, or 15.