Therapeutic Cargo: siRNA
The Proprietary siRNA Payload Behind ExoPTEN
Exosomes are being investigated as natural drug delivery vehicles because they can carry molecular cargo between cells as part of normal biological communication. This cargo may include RNA, proteins, lipids, or other defined therapeutic payloads, depending on the intended application.
NurExone’s ExoTherapy platform builds on this natural delivery function by loading exosomes with selected molecular cargo, including small interfering RNA (siRNA) sequences designed to modulate specific biological pathways. This approach supports the development of exosome-based nanodrugs that combine biological delivery properties with defined therapeutic activity.
NurExone has developed a proprietary siRNA payload designed to reduce the expression of PTEN, a protein that can restrict the regenerative response following nerve injury. The payload forms part of the Company’s broader ExoPTEN intellectual property portfolio, which includes patent protection and applications covering therapeutic cargo, drug composition and administration.
Exploring Additional RNA Targets: Perineural Network (PNN)
In addition, the Company has developed two new selective siRNA sequences that target and inhibit proteins within the Perineural Network (PNN) complex (Fig. A). The sequences, detailed in a patent application held by NurExone, are built upon a scientifically validated strategy for enhanced neuronal regeneration via inhibition of the PNN complex.
The Company believes that its innovative approach, using the Company’s ExoTherapy platform to deliver these RNA sequences, may overcome limitations of previous methods. The promise of the Company’s new sequences is evident in Figures B-C, where PNN is notably reduced post-treatment.
(A) Illustration of the extracellular Perineural network (PNN), highlighted in green.
(B) Immunohistology of one protein in the PNN in differentiated neuronal culture demonstrates the new treatment on human neuronal culture successfully degraded the PNN.
(C) Quantification of one PNN building block protein that was successfully inhibited by the new treatment.
