Exosome drug delivery technology platform

Cells communicate via exosomes, nano-sized packages of information released by  cells and absorbed by other cells in a functionally relevant manner. These packages of information contain a variety of proteins, genetic material and other cargo which have the ability to induce changes in recipient cells. Under certain conditions, exosomes produced by stem cells can be manipulated to carry different cargos. A wide variety of therapeutic molecules can be loaded into exosomes to protect the active molecule from breakdown and to target a specific area of the body. ReNeuron has therefore developed a technology by which a permanent stem cell line can be cultured  to produce specific therapeutic agents that can be harvested at a commercially relevant scale.

The ability to produce commercially valuable therapeutic products from stem cell-derived exosomes demands a standardised stem cell producer line appropriately sourced and isolated, manufactured to GMP, grown in serum-free conditions and (ideally) already having demonstrated patient safety. In the stem cell field, the CTX cell line uniquely meets all these conditions.

Summary of exosomes for therapy/ delivery vehicle:

  • Exosomes can be loaded to carry a variety of therapeutic agents for delivery to specific tissues within the body.
  • Stem cell-derived exosomes have generally been found to be less immunogenic than cells. Thus, “off the shelf” products may readily be developed using exosomes with negligible immunogenicity.
  • Exosomes can be stored without cryo-preservatives at -20°C for 6 months with no loss in their biochemical activities.
  • An important feature of exosomes is encapsulation. Protecting their contents from degradation, thereby, preventing the active ingredients from being rapidly degraded.
  • The durability of exosomes in culture permits large quantities of exosomes to be harvested and collected from cell culture medium.
  • By changing the exosome producer cell type, you can alter the region to which the payload is delivered.

The Company’s proprietary cell lines produce a panel of distinct tissue matched Exosome drug delivery candidates which have the potential to target a variety of indications and tissue types. Exosomes produced by the Company’s proprietary stem cell lines or via its induced pluripotent stem cell (iPSC) platform can be manufactured through a scalable process and loaded with a wide variety of payloads, such as nucleic acids (including siRNA, mRNA and miRNA), proteins (such as Cas9, antibodies and peptides) as well as small molecules.

ReNeuron has signed a number of collaboration agreement with major biopharmaceutical companies and academic institutions to explore the potential of our exosome technology to deliver novel therapeutic agents to the brain and other regions of the body. Positive pre-clinical data from an ongoing collaboration with the University of Salamanca, Spain, has already provided proof-of-concept that ReNeuron’s novel Exosome drug delivery technology can effectively deliver therapeutic proteins to the specific region of the brain affected by several neurological diseases such as stroke, Parkinson’s disease and Huntington’s disease. These studies have shown that ReNeuron’s Exosome technology offers higher stability, more targeted delivery, and an increase in potency, therefore potentially solving the delivery issues that can be experienced with therapeutic proteins.

This could be of interest to major pharmaceutical companies to aid in the treatment of a variety of neurological diseases.