NurExone Biologic Ltd., an Israeli biopharmaceutical company aiming to revolutionize the treatment of spinal cord injury (SCI), is leveraging its proprietary technologies into a platform technology company with a potentially diverse portfolio of novel, cutting-edge therapies for several CNS traumatic indications.

The global acute Spinal Cord Injury (SCI) therapeutics market is expected to reach US$6.6 billion by 2028, when North America has the largest market share. Currently, 143 Exosome-based clinical trials are running. Exosomes based therapies are considered the next generation therapy when it comes to SCI.

Exosomes are nano-sized vesicles that cells naturally produce into extracellular fluids upon the fusion of multivesicular bodies and the plasma membrane. Exosomes are 30-150 nm in size and encapsulate a variety of biomolecules in a phospholipid-bilayer membrane, capable of transferring DNAs, microRNAs, non-coding RNAs, and lipids between cells with or without direct cell-to-cell contact, acting as intercellular messengers. It was shown that loaded exosomes can deliver biomolecules into target cells, where they unload their therapeutic cargo, creating a healing environment and altering biological response. Because of their small size, they can transport critical signaling proteins and genetic data from cell to cell, which can help with a variety of conditions, and which can form the basis for many therapies, including Central Nerve System (CNS) as can pass the Blood Brain Barrier (BBB) and penetrate the brain and Spinal Cord. 

NurExone holds key proprietary technologies that uniquely position the company as a potential leader in the exosome technology field: large-scale exosome production, distinctive small-interfering RNA ("siRNA") sequences as the exosomes' cargo, and the loading technology used to load therapeutic cargo into exosomes to achieve a therapeutic effect. In addition, the company is developing in a co-joint project a non-invasive technology for intranasal administration of exosome-based therapies. In scientific research, the company found its large-scale exosome production process effective and recorded the efficiency of its proprietary siRNA sequences as a potential therapeutic cargo. The company also conducted a pre-clinical study of its technology for loading therapeutic cargo into exosomes and reported positive results. On December 2, the company released findings indicating that loading therapeutic molecular cargo into exosomes may be accomplished effectively using its novel and exclusive loading technology. These exosomes will be biologically directed to a specific damaged location that needs healing, where they will "dock" and unload their therapeutic cargo into the neuronal cells. The findings of the study showed that the company's proprietary loading technology achieved the desired loading efficiency and that NurExone's loading moiety - a fragment of a molecule - does not impair the ability of the siRNA to knock down gene expression., validating NurExone's technology while also vastly expanding their market offering. 

Additionally, NurExone has created and presented proprietary loading technology that addresses the exosomes industry's bottleneck. In October, the company released Productivity and Quality Pilot Results of a Patent-Pending Process for three-dimensional (3D) Scaled Up Exosome Production, demonstrating the potential of its novel and exclusive production process to increase exosome yields while also improving the potency of neuron regeneration and resulting in cost savings in production. The development of scaffolds intended to control various aspects of the tissue formation process has received a lot of interest in recent years. The use of the 3D method in the field of regenerative medicine holds great promise for the repair and reconstruction of various anatomical defects in complex organs and functional tissues while encouraging cell attachment, proliferation, and extracellular matrix production. 3D Scaffolds are porous biomaterials designed to allow the transport of body liquids and gases, promote cell interaction, viability, and extracellular matrix (ECM) deposition, and bio-degrade at a controlled rate.

In a pilot study, the company compared the productivity and quality outcomes of the shear stress production process using current two-dimensional (2D) and cutting-edge 3D production techniques by evaluating human bone marrow mesenchymal stem cells (hBM-MSC) and dental pulp stem cells (DPSC). The findings demonstrated that shear stress significantly increases exosome secretion from stem cells grown on a 3D scaffold.Once the production process is complete, NurExone intends to monetize this technology by licensing it to other biopharmaceutical companies as well as incorporating it into its exclusive drug platform.

The company's first under development product, leveraging the capabilities of its advanced platform will be ExoPTEN for spinal cord injuries. ExoPTEN an exosome-based non-invasive therapy administered intranasally, is expected to aid in nerve regeneration, allowing neural activity to be restored and spinal cord damage to be reversed, effectively curing SCI. These are also being actively researched for the treatment of cancer and neurological diseases. NurExone has the potential to become a market leader in exosome-based SCI treatment, and its successful milestones as of now are opening the door for its technology to be used in development of other treatments/indications.