RENE.L Regulatory News (RENE)

ReNeuron Group plc

("ReNeuron" or the "Company")

Exosome platform update

Further collaborations underway to demonstrate the potential of ReNeuron's proprietary exosome platform

Emerging data shows platform's application to a range of novel therapeutics targeting the brain and other tissues

ReNeuron Group plc (AIM: RENE), a UK-based global leader in the development of cell-based therapeutics, provides an update on the positive progress being made by third party commercial collaborators with its exosome technology platform and the signing of new collaboration agreements with a major pharmaceutical company and leading academic institutions in the UK and mainland Europe.

About ReNeuron's exosome technology

ReNeuron's exosome technology is being explored by pharmaceutical, biotechnology and academic collaboration partners as a novel delivery vehicle for third party therapeutic agents targeting the brain and other parts of the body. The Company is developing its lead exosome candidate from its proprietary CTX neural stem cell line. These exosomes can be manufactured according to GMP standards through a fully qualified, xeno-free, scalable process and loaded with a diverse range of therapeutic agents, including siRNA/mRNA/miRNA, CRISPR/Cas9, antibodies, peptides and small molecules. The exosomes have been shown to exhibit a natural ability to cross the blood brain barrier.

ReNeuron is also developing further exosome candidates derived from a panel of additional producer cell lines owned by the Company. These exosome candidates have the potential to broaden the repertoire of tissues and indications that the Company is able to target.

Further collaboration agreements

ReNeuron is exploring multiple methods of loading exosomes, both internally and on the exosome surface, and is collaborating with major pharmaceutical/biotechnology companies on these projects. Since the last update in the Company's interim results statement on 24 November 2020, the Company has signed a further commercial collaboration agreement with a major pharmaceutical company, focusing on the potential of the Company's exosomes to deliver DNA cargoes for expression of therapeutic genes in the brain. 

A further two collaborations have also been initiated with leading academic institutions in the UK and mainland Europe, focusing on the delivery of CNS-targeting growth factors and siRNA to the brain. ReNeuron has demonstrated engagement of target receptors in the CNS by exosome-loaded growth factors during a recent pilot study. Ongoing work in these collaborations will aim to consolidate this finding, leading to further studies examining functional delivery of the loaded exosomes.

Positive early pre-clinical data

Since the last update on 24 November 2020, the Company has shown highly efficient loading of nucleic acid payloads in its exosomes and these exosome candidates have also demonstrated functional payload delivery, both in vitro and in vivo, to the brain and peripheral tissues via repeat-dose intravenous administration.

Specifically, target knockdown by exosome candidates was assessed in multiple brain regions and in key peripheral tissues including heart, kidney and skeletal muscle. Evidence of target knockdown was observed in each of these organs suggesting these exosomes have the potential to deliver payloads to therapeutically-meaningful levels to a variety of tissues. These studies also suggest the loaded-exosomes are well-tolerated, laying the foundation for expansion to functional delivery studies.

In addition to exploiting natural exosome tissue specificity, ReNeuron has also now successfully decorated the surface of its neural stem-cell derived exosomes with a specific tissue-targeting peptide. This proprietary peptide was modified to enhance binding to the exosome surface, resulting in a 10-fold increase in surface binding compared with unmodified peptide. The next phase of this collaboration aims to confirm that the peptide promotes exosome targeting to additional tissues in vivo. This peptide platform has the potential to generate further targeting peptides to that would rapidly expand the therapeutic reach of ReNeuron's exosome candidates.

Further data across these collaborations are expected during the course of the next six months which, if positive, will enable subsequent potential out-licensing deals with the Company's exosome platform.

Olav Hellebø, Chief Executive Officer, commented: "Our exosome platform is being deployed in collaboration with commercial third parties and it is pleasing to report that these collaborations are progressing to plan, with exciting data now emerging to demonstrate the delivery potential of this technology with a range of novel therapeutic agents, targeting the brain and other tissues. We look forward to reporting further progress across these collaborations in the months ahead."

This announcement contains inside information. The person responsible for arranging for the release of this announcement on behalf of the Company is Olav Hellebø, Chief Executive Officer.

ENDS

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About ReNeuron

ReNeuron is a global leader in cell-based therapeutics, harnessing its unique stem cell technologies to develop 'off the shelf' stem cell treatments, without the need for immunosuppressive drugs. The Company's lead cell therapy candidate is in clinical development for the blindness-causing disease, retinitis pigmentosa.

ReNeuron is also advancing its proprietary exosome technology platform as a potential delivery system for drugs that treat diseases of the brain. The Company also has the ability through its conditionally immortalised induced pluripotent stem cell (iPSC) platform to make any tissue cells of choice; in-house programmes are focused on treatments for blood cancers and diabetes.

ReNeuron's shares are traded on the London AIM market under the symbol RENE.L.

For further information visit www.reneuron.com