NurExone Biologic Inc., a leading biotechnology company, has recently achieved a significant milestone in the development of treatments for acute spinal cord injuries.
The U.S. Food and Drug Administration (FDA) has granted Orphan-Drug Designation (ODD) to the company’s innovative ExoPTEN therapy. This designation is crucial for conditions like acute spinal cord injury, where effective treatments are currently limited.
The orphan drug designation is particularly beneficial for pharmaceutical companies focusing on rare diseases, defined as those affecting fewer than 200,000 people in the United States. It offers several advantages, including market exclusivity, financial incentives, regulatory assistance, and support in drug development. These benefits are designed to encourage the development of treatments for less common conditions, thereby improving access to therapies for patients who need them.
ExoPTEN therapy, developed by NurExone, involves the use of mesenchymal stem cell (MSC) derived small extracellular vesicles (EVs). These are loaded with short and modified interfering RNA (siRNA) targeting the phosphatase and tensin homolog (PTEN) protein, specifically for treating acute spinal cord injuries. The therapy is currently under development and has shown promise in its potential impact on patients suffering from these injuries.
Dr. Lior Shaltiel, CEO of NurExone Biologic, expressed optimism about the orphan-drug designation’s role in streamlining the company’s go-to-market strategy, shortening the regulatory process, and providing market exclusivity. This recognition by the FDA is a testament to the potential impact of ExoPTEN therapy on the lives of patients with acute spinal cord injuries.
NurExone Biologic Inc. is a TSXV listed pharmaceutical company developing a platform for biologically-guided ExoTherapy. This therapy is intended to be delivered non-invasively to patients who have suffered traumatic spinal cord injuries.
The treatment concept was initially demonstrated in animal studies at the Technion, Israel Institute of Technology. Through its fully-owned subsidiary, NurExone Biologic Ltd, the company is now translating this treatment for human use.
NurExone holds an exclusive worldwide license from the Technion and Tel Aviv University for the development and commercialization of this technology.
For more information about NurExone Biologic Inc. and their groundbreaking work in spinal cord injury treatment, interested parties can visit their website or follow them on various social media platforms.
The press release also contains forward-looking statements regarding the potential of ExoPTEN and its benefits. These statements are based on current expectations and projections about future results. However, they involve significant risks, uncertainties, and assumptions, including the company’s stage of development, market acceptance, technological changes, dependence on key personnel, and other factors. These risks are detailed in the company’s Annual Information Form and should be considered carefully. The forward-looking statements are made as of the date of the press release, and the company assumes no obligation to update or revise them except as required by law.