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Publication of Pre-Clinical Data Relating to ReNeuron’s Exosome Nanomedicine Platform

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Publication of Pre-Clinical Data Relating to ReNeuron’s Exosome Nanomedicine Platform

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ReNeuron Group plc has announced the publication in a peer-reviewed scientific journal of important characterization data relating to its exosome nanomedicine platform.

ReNeuron is researching the therapeutic potential of exosomes derived from its proprietary stem cell lines. The Company is also exploring the potential of its exosomes as a delivery system for gene therapy treatments. Exosomes are lipid-based nanoparticles secreted from all cells. They play a key role in the transfer of beneficial proteins and particularly non-coding microRNAs (miRNAs) from one cell to another.

The paper, “Investigation of content, stoichiometry and transfer of miRNA from human neural stem cell line derived exosomes”, has been published in the Journal PLOS ONE and describes work undertaken by ReNeuron researchers to characterize the composition of exosomes secreted from the Company’s clinical-grade CTX stem cell line. The paper identifies a unique set of highly enriched miRNAs contained within CTX-derived exosomes. The exosomes were able to transfer these miRNAs out of CTX cells to neighbouring cells, where they significantly down-regulated the expression of a target protein within the recipient cells.

The Company believes that this is the first published molecular demonstration that exosomes can deliver a therapeutic microRNA in sufficient quantities to alter protein expression in target cells. In particular, miRNAs delivered via ReNeuron’s CTX-derived exosomes may have significant impact in the regulation of target cell growth and cell death.

The paper therefore lends empirical support to a potential miRNA-based mechanism of action of CTX-derived exosomes as a therapeutic agent in cancer applications. The data also point to the potential use of CTX-derived exosomes as a delivery vehicle for other therapeutic genes in gene therapy applications.

The PLOS ONE paper can be found at:
http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0146353

ReNeuron has filed multiple patent applications covering the composition, manufacture and therapeutic use of its exosome nanomedicine platform, including the particular miRNAs that are highly expressed and their therapeutic use in cancer. The Company has an ongoing programme of work to investigate the mechanism of action and utility of its exosome nanomedicine platform in a range of potential cancer indications.

The publication of the PLOS ONE paper follows yesterday’s announcement by ReNeuron that it had been awarded a £2.1 million (US$3.0m) grant from Innovate UK to further advance its exosome nanomedicine platform in collaboration with the Cell Therapy Catapult and the Department of Biochemical Engineering at University College London.

Dr John Sinden, Chief Scientific Officer of ReNeuron, said: “This paper demonstrates the unique characteristic of our CTX cells to preferentially enrich a set of microRNAs contained within their exosomes, which we have shown to have anti-cancer properties in pre-clinical models. The paper therefore represents a significant milestone in ReNeuron’s ongoing research to harness the potential of exosomes derived from its proprietary stem cell lines as therapeutics in their own right and also as a system for delivering therapeutic genes.”

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