Israeli biotech startup developing innovative platform for treatment of nervous system diseases

  (photo credit: INGIMAGE)
(photo credit: INGIMAGE)

With 250,000 to 500,000 people sustaining Spinal Cord Injury (SCI) around the world yearly, Israel-based biotech startup NurExone Biologic is developing a revolutionary biological Extracellular Vesicles (EVs)-based technology drug platform to provide functional recovery from damage in the central nervous system (CNS). 

The company is developing treatment for full or partial reversal of paralysis and improving the lives of patients following spinal cord injury using bio-guided exosomes (membrane-bound extracellular vesicles) loaded with modified small interfering RNA (siRNA) sequences. NurExone’s technology is being used to develop treatment of various conditions, including spinal cord injury (SCI), traumatic brain injury, and other CNS indications. An animal study demonstrated that the breakthrough exosome-based technology has immense potential to treat SCI. Following this study, NurExone was granted an exclusive worldwide license from the Technion – Israel Institute of Technology and Tel Aviv University, both located in Israel, to develop and commercialize this technology.

According to the National Cancer Institute, exosomes which look like tiny sacs approximately 30 to 150 nm in diameter are formed inside a cell and contain some of the cell’s proteins, DNA, and RNA. Exosomes are generated and released into the blood of all living systems by many cell types, traveling through the blood to other parts of the body. EVs are generated by many cell types and contain not only proteins and lipids but also mRNAs and microRNAs (miRNAs). 

NurExone’s R&D team, which consists of PhD-scientific researchers with proven track records and extensive expertise, are advancing in their research for both the specific treatment of spinal cord injuries as well as the overall technology which should serve as a platform for numerous other treatments. 

Why is this important?

Damage to the CNS causes a long and complex chain of events following the injury itself which can affect the efficiency of treatments. Due to the lack of plasticity and limited regenerative capacity of the CNS, recovery of neural function after SCI is rare. Following injury, the spinal cord goes through a number of cellular and molecular alterations that prevent axonal regeneration. As a result, about 5.4 million people, or 1.7% of the U.S. population live with some type of paralysis, with spinal cord injuries causing 27.3% of those cases.

Recent advances in the medical management of SCI have significantly improved the diagnosis, stabilization, survival rate, and well-being of SCI patients. However, there’s a need for more efficient treatment that could reverse paralysis or cause functional recovery. To meet this need, considerable efforts are being made by SCI researchers. While companies like Abbvie, Kringle pharma, Eusol, and Evox Therapeutics are already in the business of treating people with spinal cord injuries, NurExone is uniquely disrupting the exosome space — a market estimated at approximately $US 3 billion by 2030. For many years, Israel’s biotech industry has been perceived as less promising than the thriving medtech scene; however, that narrative is now changing quickly. The country’s biotech space has seen significant innovations and attracted more investments recently, and companies like NurExone are leading the race in an ecosystem bustling with strong momentum.

“Currently, no available treatment technologies has all four of our product’s properties - potential to repair full transection, non-immunogenic, off-the-shelf use, and non-invasive,” said CEO Dr. Lior Shaltiel, who added that “exosomes have emerged as promising nanocarriers for drug delivery and targeted therapy for SCI, as an alternative to stem cell therapy.”

“For tiny functional molecule delivery, EVs are very well-suited. and increasing evidence suggests that they also play a crucial role in cell-to-cell communication. EVs are promising therapeutic agents because their complex cargo of proteins and genetic materials has diverse biochemical potential to participate in multiple biochemical and cellular processes, an important attribute in the treatment of complex diseases with multiple secondary injury mechanisms involved, such as traumatic brain injury (TBI) and spinal cord injury (SCI),” Shaltiel added.

A deeper look into NurExone’s mission – SCI and beyond

Founded in 2020 by world-class researchers, Prof. Shulamit Levenberg and Prof. Daniel Offen, NurExone’s aims to change the way spinal cord injury (SCI) is treated around the world by utilizing exosome technology to deliver treatment to patients via a non-invasive delivery platform. The treatment will be administered through the nose which would have a natural effect in targeting neuron damage. While many companies are using stem cells that release exosomes naturally and attempt to regenerate neurons through local injections, Shaltiel said that loaded exosomes transferred into the body nasally would be a considerable game-changer for the millions of patients who suffer this type of injury.

According to Shaltiel, compared to other forms of treatments, EVs are more amenable to development as an “off-the-shelf” therapeutic agent that can be delivered to patients in a timely manner. He further explained that exosomes, which are not cells, do not have “the risks associated with delivery of viable cells such as occlusion (blockage of an opening or blood vessel) in the microvasculature or unregulated growth of transplanted cells.”

While stem cell therapy has attracted great attention as a treatment strategy for SCI, Shaltiel said NurExone’s technology has the potential to vastly impact other indications as the company is developing a technology with higher therapeutic potential which promises an effective technique for loading therapeutic agents into the exosomes. 

Moreover, Shaltiel noted that, “NurExone is developing an exosome-based drug that has already been demonstrated with a Proof of Concept (POC) on rats for acute spinal cord injury. We look forward to translating the success in rats to future clinical trials in humans.”  To expand its reach and improve its drug administration, the company is currently in collaboration with leading pharmaceutical companies such as Polyrizon, Denovomatrix, and Nanometrix.

In a market that’s received approximately $US 570 million in investments since 2017, according to Roots Analysis, Shaltiel believes the business potential is significant. “Scientifically, we see an increased demand for exosome therapy, which is considered a ‘next generation’ therapy for CNS indications,” he said.

This article was written in cooperation with Future Markets Research