ReNeuron’s stem cell derived proprietary Exosome Technology platform offers a delivery mechanism for a variety of payloads such as siRNA, mRNA, proteins, small molecules and genes. The Company has a growing number of partner collaborations with Global Pharma, Biotech and academic partners in this fast-expanding area of scientific and commercial interest. ReNeuron also has the ability through its conditionally immortalised induced pluripotent stem cell (iPSC) platform to make allogeneic tissue cells of choice and has the potential to produce exosomes with tissue specific targeting ability.
The Company has two clinical stage stem cell therapy candidate assets, a Phase 2a human retinal progenitor cell (hRPC) therapy candidate in retinitis pigmentosa and a phase 2b immortalised neural progenitor cell therapy candidate (CTX) in stroke. Both these programmes are out-licensed to Fosun Pharma in the Peoples’ Republic of China (China) and are now also available for out-license outside of China.
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Exosome technology platform
The Company’s proprietary cell lines produce a panel of distinct tissue matched Exosome drug delivery candidates which have the potential to target a variety of indications and tissue types. Exosomes produced by the Company’s proprietary stem cell lines or via its induced pluripotent stem cell (iPSC) platform can be manufactured through a scalable process and loaded with a wide variety of payloads, such as nucleic acids (including siRNA, mRNA and miRNA), proteins (such as Cas9, antibodies and peptides) as well as small molecules.
Induced pluripotent stem cell (iPSC) platform
Pluripotent stem cells are cells that have the capacity to self-renew and to develop into the three primary germ cell layers of the early embryo and therefore into any cell type. Induced pluripotent stem cells or iPSCs are reprogrammed adult cells that have the same capacity. New data has shown that ReNeuron’s proprietary human neural stem cell line (CTX) can be readily reprogrammed to pluripotency while maintaining its conditional immortalisation technology. Allowing the rapid development of new allogeneic cell lines as cell-based therapeutics or for the production of exosomes with specific tissue targeting.
Our human retinal progenitor cell line (hRPC) has been developed for the treatment of retinal diseases. Clinical development of hRPC for retinitis pigmentosa, a blindness-causing disease of the retina, has been ongoing in the US in a Phase 1/2a clinical study and there is potential to expand the hRPC platform to target further retinal diseases such as cone-rod dystrophy. The Company intends to out-license its RP programme assets (outside of China) following completion of the current clinical data package.
ReNeuron’s immortalised neural progenitor cell line (CTX) is being developed as a therapy for the treatment of patients left disabled by a stroke. The company has previously announced positive Phase 2 data with this therapeutic candidate and clinical development was approved to progress into a randomised, placebo-controlled Phase 2b clinical trial in the US. Following the strategic decision to terminate this Phase IIb study, this programme will continue through its partnership with Fosun Pharma in China and is available for out-licence through partnerships outside of China.
Ultimately, we expect to realise value for our technologies and therapeutic programmes via out-license to commercial development partners at the appropriate points in their development. We have partnered with Shanghai Fosun Pharmaceutical Industrial Development Co., Ltd. for the development, manufacture and commercialisation of our hRPC and CTX programmes in China and are seeking other parties to develop those programmes outside of China.
In order to achieve these objectives, we work closely with a number of key academic and industrial partners while continuing to maintain tight control over our financial resources.
ReNeuron is incorporated in the UK with offices in the UK and the US. Our shares are traded on the London AIM market under the symbol RENE.L