The Product
Born in the labs of Israel’s Technion, ExoPTEN is NurExone’s first nanodrug. Being developed for patients who have suffered acute spinal cord injury. ExoPTEN uses exosomes loaded with a specific and proprietary sIRNA sequence as the active pharmaceutical ingredient. Studies have demonstrated that ExoPTEN facilitates nerve regeneration, regrowth, and functional recovery following a brief intranasal administration in laboratory animals.
Roadmap to Success
Control Group
Treated With ExoPTEN
Preclinical Studies
The regeneration of neurons and motor restoration in rats has been successfully replicated across multiple experiments conducted in NurExone’s laboratories.
These results hold significant importance particularly when considering the challenges associated with reproducibility in scientific research. They both validate our scientific approach and instill confidence in our ability to translate these findings into tangible benefits for human patients
Motor function, sensation and bladder control recovery occurred in 75% of animals following a short, intranasal ExoPTEN cycle.
See for yourself
Potential Benefits
Competition
ExoPTEN v. Other Therapies Under Development for Acute Spinal Cord Injury
Healthcare Burden
Spinal Cord Injury
ExoPTEN is targeted at patients who have recently suffered an acute spinal cord injury, e.g., vehicle, work, sports-related injuries.
Globally, an estimated 250,000–500,000 people suffer from spinal cord injuries (SCIs) annually, with 90% of these injuries stemming from traumatic causes such as vehicle accidents, workplace incidents, or sports-related mishaps. In the United States alone, this accounts for approximately 17,000 new cases per year, while in Europe, there are around 10,000 new cases annually. This suggests a potential market for ExoPTEN of approximately 50,000 new cases per year.
The financial burden on both patients and the healthcare system is considerable. It involves immediate expenses, such as emergency surgeries, followed by prolonged rehabilitation periods. Additionally, many individuals with spinal cord injuries require ongoing financial support to accommodate various disabilities and cope with unemployment.
Spinal cord injury care – ~90% of new cases each year are from trauma
The Science of ExoPTEN
How does ExoPTEN Work?
siRNA for PTEN Inhibition
Axonal growth and functional recovery following SCI are limited, because of the poor innate regenerative capacity of adult central nervous system neurons and the hostile injury environment comprising inflammation, myelin- associated inhibitors, glial scar components and compromised blood supply.
PTEN is expressed in neurons and regenerating axons and plays a vital role in controlling the regeneration of corticospinal neurons via downregulating cytoplasmic mammalian target of rapamycin (mTOR) activity.
This mTOR pathway is inhibited in axotomized adult neurons, restricting protein synthesis to sustain axonal growth. This unique activity makes PTEN a major homeostatic regulator and tumor suppressor protein, which function is absent or defective in a wide variety of tumors as a result of somatic alterations. The important role of the PI3K/AKT/mTOR signaling pathway in cell growth, regeneration and survival supports the rationale for the therapeutic targeting of PTEN.
Among the suggested PTEN inhibition-based therapeutic targets are nerve growth and regeneration after injury or damage, treatment of cardiac ischemia/reperfusion and associated disease, wound repair, and infertility.