Exosome system that utilizes miRNA to selectively target cells or tissues for the delivery of therapeutic nucleic acid sequences.

The Need

Typically, over 90% of a therapeutic drug is wasted in the body, leading to unwanted side effects for the patient (marketesearch.com). For this reason, there has been a large focus on the improvement of drug delivery systems through encapsulation and tissue targeting. One method developed for targeted drug delivery involves the use of oligonucleotides to provide the targeting function of the system. Though oligonucleotides have a promising ability to target specific tissues, there are still problems with the clinical development of these agents. The size and charge of oligonucleotides cause them to be unstable in circulation, and make it difficult for oligonucleotides to penetrate the cell membrane. Additionally, the current gold standard method for oligonucleotide delivery involves the use of lipid nanoparticles. These are composed of synthetic ingredients that can decompose in vivo to produce cytotoxic or immunogenic effects. In order to produce a targeted oligonucleotide drug delivery system that is clinically effective, these problems need to be addressed.

The Market

  • The global market for nucleic acid aptamers reached $340.5 million in 2014. This market should grow to roughly $610.3 million in 2015 and about $5.4 billion in 2019. (BCC Research)
  • The global revenue for advanced drug delivery systems totaled $151.3 billion in 2013. In 2018, revenues are estimated to reach nearly $173.8 billion. (BCC Research)
  • During the past three decades formulations that control the rate and period of drug delivery (i.e. time-release medications) and target specific areas of the body for treatment have become increasingly common and complex. (BCC Research)
  • According to MarketResearch.com, nanocarriers will account for 40% of a $136 billion nanotechnology-enabled drug delivery market by 2021.

The Technology

The Ohio State University researchers, led by Dr. Thomas Schmittgen, developed an exosome drug delivery system that targets specific cells or tissues through miRNA. This technology incorporates a primary nucleic acid sequence that is used to target the exosome to a specific cell or tissue. The secondary nucleic acid sequence is encapsulated within the exosome and encodes for the therapeutic polynucleotide. One unique feature of this technology is that there is no need for synthetic oligonucleotides. The therapeutic nucleic acid cargo is synthesized by the cells that produce the exosomes, thus reducing the cost of the exosome device. This technology is currently being developed for the treatment of hepatocellular carcinoma cells and tumors, but could be applied to other cancer types in the future.

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