Exosomes loaded with siRNA against PTEN, developed at the Technion and Tel Aviv University, are designed to reduce inflammation and encourage regeneration in the central nervous system – an area that is also relevant to myelin damage such as in multiple sclerosis.
Dr. Lior Shaltiel, CEO of NurExone since 2021, describes the company as developing regenerative therapies based on exosomes – tiny biological "messengers" that the body uses to communicate between cells and tissues. These are natural nanoparticles with intercellular communication capabilities and regenerative properties, which can also be harnessed to deliver therapeutic "cargo" directly to damaged tissue.
The company is a spin-off of the Technion and Tel Aviv University, based on joint research by Prof. Shulamit Levenberg of the Technion and Prof. Danny Offen of Tel Aviv. The two researchers have been working since 2015 on a project aimed at the regeneration of neurons in the central nervous system, an area where the body has difficulty renewing nerve cells and restoring full function after trauma.
How the ExoTherapy platform works
At the heart of the development is a platform called ExoTherapy, which aims to “load” exosomes with therapeutic molecules and turn them into a targeted drug. Exosomes are created when stem cells proliferate, and they exhibit the ability to “homing” – preferentially reaching certain tissues and foci of inflammation. The company uses them as a kind of “guided missiles,” which can be loaded with a variety of therapeutic payloads.
The leading product is called ExoPTEN, and is based on exosomes that carry a siRNA (short RNA that silences gene expression) payload that suppresses the expression of the PTEN protein – a biological target studied in the context of nerve growth and regeneration. According to Shaltiel, the exosomes work through two parallel mechanisms: reducing inflammation in the area of injury and encouraging natural regeneration of nerve tissue.
The “no living cells” approach is expected to reduce risks associated with cell transplants, such as immune rejection. In addition, the company claims that exosomes can cross the blood-brain barrier and reach sites of injury. Another feature is the possibility of producing an “off-the-shelf” product – easier to manufacture, distribute and administer, compared to treatments that require a personalized process for each patient.
Initial applications include spinal cord injury, optic nerve injury, peripheral nerves, and traumatic brain injury (TBI). In preclinical studies, the company has used rat models of complete spinal cord transection, with intranasal (through the nose) or intrathecal injection.
Why can they "suddenly" sell the base of the technology as well?
The important business point is that it is not just about one future drug, but also about the ability to produce the "basic material": "naive" exosomes (i.e., before loading with a dedicated drug payload). Norexon is establishing a US subsidiary called Exo-Top to produce "therapeutic-grade" exosomes from a unique Master Cell Bank of mesenchymal stem cells (MSCs).
Here comes the explanation for the ability to "sell the base": the same production line that serves the drug pipeline can also produce exosomes that are not defined as a specific drug, but as a product for non-pharmaceutical uses. The company presents this as a "dual use" model: the exosomes produced in Exo-Top are intended to serve both as a carrier for the therapeutic pipeline, and also to feed a rapidly growing "regenerative exosomes" market in the fields of aesthetics, dermatology, pain medicine and longevity.
Simply put: In regular biotech, revenue comes only after clinical trials and regulatory approval. Here, the company is trying to generate early revenue from selling high-quality “biological raw material” before the drug itself reaches the market. The company is targeting the start of commercial sales in 2026.
Shaltiel points to a significant regulatory change in the US: As of July 2025, the state of Florida announced that cell and exosome-based therapies can be performed for three indications (pain, orthopedics, and wound healing) without a full FDA clinical trial, solely based on toxicity tests. Utah and Nevada have also taken similar steps, in what is seen as part of an effort to encourage regenerative therapies in the US instead of American citizens flying to Central America or the Far East to have these treatments performed.
What's next: IND and first-in-human trial
The company is in the preclinical stage and is working on preparing an IND (Investigational New Drug) file with the FDA. According to Shaltiel, the goal is to begin clinical trials in humans in early 2027. The company is also considering submitting applications for compassionate use in Israel, especially for patients with spinal cord injuries or severe glaucoma who have no alternative treatment.
It is important to emphasize: therapeutic exosomes of this type are not yet an approved treatment for routine use in spinal cord injuries or glaucoma; these are development directions based on preclinical research and regulatory planning.
The stock market and financing aspect
Norexon has been a public company traded on the Canadian Stock Exchange since June 2022. Shaltiel describes the choice to go public at an early stage as a "disruption" in the way biotechs are financed. Biotech companies typically operate as private companies until a significant financing event or exit, but Norexon chose the public path early on.
The advantage, he claims, is that investors can liquidate their investment after a lock-up period of just four months, instead of waiting years for an exit event. "An investor who loves biotech and knows he can liquidate the money after a short period and not wait years, that's a very significant thing," he explains.
The company's last funding round was in November 2025, when it raised $3.2 million through warrants from existing investors. The company's investors are primarily private investors and wealthy families (Family Offices), rather than traditional venture capital funds. The company plans to move to a US stock exchange (NASDAQ or NYSE) in the near future.
About 86% of the money raised through the Canadian company goes to Israel. The company employs 22 people, most of them in Haifa and the north of the country, and operates out of the Technion as part of the university's spin-off companies. Some of the patents belong to the company, and some are under exclusive license from the Technion and Tel Aviv University.
Shaltiel emphasizes the importance of financing Israeli biotech companies: “We have two problems in Israel: we lack money and we lack human capital. Or we could have good human capital that would do industry, not just science.” He also directs an MBA program in biomedical management at the Hebrew University, which aims to train the next generation of management in the Israeli biotech industry.
The business environment of recent years has been particularly challenging for biotech companies. He says, “If you used to be able to get an investment every five meetings, now you’re getting an investment every 20 meetings.” He notes that companies that think “outside the box” with financing – such as crowdfunding, crypto financing, or hybrid models – are the ones that have managed to survive the crisis.
warning: The information in this article is for informational purposes only and does not constitute medical or investment advice. Exosome-based therapies are still in the research and development stages, and are not approved for routine clinical use in most indications.
More of the topic in Hayadan:
Dr. Lior Shaltiel will present at the conference NANO.IL International To be held at the Nation Buildings in Jerusalem (March 8-10). The conference will showcase the best Israeli scientists and entrepreneurs who have taken nanotechnology and integrated it into groundbreaking applications in a variety of fields: security, medicine, industry, chemistry, and more.
