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ExoPTEN is an investigational exosome-based therapy developed by NurExone Biologic for the treatment of acute spinal cord injury and other central nervous system injuries. The drug utilizes mesenchymal stem cell-derived extracellular vesicles (exosomes) loaded with a proprietary small interfering RNA (siRNA) sequence targeting PTEN, a key protein that inhibits nerve regeneration. By silencing PTEN expression, ExoPTEN aims to promote nerve regeneration, regrowth, and functional recovery following injury. Preclinical studies have demonstrated significant improvements in motor function recovery and blood flow at the site of spinal cord injury in animal models after intranasal administration. Additionally, ExoPTEN has shown potential for restoring optic nerve function in preclinical models simulating glaucoma and other optic neuropathies. The therapy has received Orphan Drug Designation from both the US FDA and European Medicines Agency for acute spinal cord injury[1][2][3][4][5][7].
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