Exosome platform: ExoPTEN as a novel therapy for nerve regeneration, and beyond

Introduction:

NurExone Biologic pioneers exosome-based therapeutics to promote nerve regeneration across various clinical indications. Our lead candidate, ExoPTEN, employs bone marrow mesenchymal stem cells derived exosomes to deliver a proprietary siRNA targeting PTEN, a key regulator of the mTOR signalling pathway. By reducing PTEN expression, ExoPTEN promotes neuronal survival, axonal growth, and regenerative capacity. Preclinical efficacy was successfully demonstrated in the rat models of spinal cord injury (SCI) and optic nerve crush (ONC).

Methods:

Naïve exosome’s identity was detected by Western Blot and F-NTA Tetraspanin Detection Kit; potency was assessed by CD73 activity and immunomodulation. ExoPTEN ability to reduce PTEN was demonstrated by RT-qPCR method.

Therapeutic efficacy in spinal cord injury (SCI) models was evaluated using MRI, locomotor scoring, CatWalk XT gait analysis, von Frey sensory testing, and immunohistochemistry. In the ONC model, retinal ganglion cell (RGC) survival and function were evaluated by histology and electroretinography.

Results:

3D manufacturing scale demonstrated high robustness of the generated exosomes, assessed via size, yields, purity, surface markers and potency across batches, supporting reliable production for future clinical applications. ExoPTEN treatment effectively reduced PTEN expression in vitro, resulting in significant therapeutic efficacy in preclinical models.

Conclusion:

Our scalable and robust manufacturing capabilities ensure high product consistency, driving the clinical progress of exosome-based therapies. These technological foundation paves the way for future clinical application of naïve exosomes and ExoPTEN, a promising therapeutic strategy for treating SCI and other types of CNS injuries.