Using our unique and scaleable stem cell technologies, we have created a pipeline of commercially focused stem cell therapy candidates addressing significant areas of unmet medical need. These therapeutic candidates are based around two core stem cell assets, our CTX neural cell line and our human retinal progenitor cells (hRPC). Our exosome platform is yielding encouraging early pre-clinical data accross a range of potential indications which are being investigated further.
We have used our cell expansion and screening technologies to develop “off-the-shelf” stem cell therapies for serious conditions such as stroke where the patient populations are significant and where few if any alternative treatments exist. Unlike conventional drug treatments which typically address the symptoms of disease, the potential of stem cell treatments such as ours is to address the underlying causes of the target disease. Our stem cell treatments have been shown in pre-clinical testing to stimulate natural repair mechanisms in the organs affected by the disease in question, leading to a reduction in the functional impairments associated with the disease.
Our lead CTX stem cell therapeutic candidate is a therapy for the treatment of patients left disabled by the effects of a stroke. Our second application for the CTX cells is for the treatment of critical limb ischaemia. Both treatments are currently in clinical development. Our hRPC stem cell candidate is for the treatment of retinitis pigmentosa, a blindness-causing disease of the retina. This treatment is in late pre-clinical development. The characteristics of the CTX cell line give ReNeuron some distinct technical and competitive advantages in the field. The CTX cell line has been taken through a full manufacturing scale-up and quality-testing process. As such, it represents a standardised, clinical and commercial-grade cell therapy product capable of treating all eligible patients presenting with the diseases targeted, without the need for additional immunosuppressive drug treatments. There will be no need to re-derive and test new cell lines for subsequent clinical trials or for the market – all such cells can simply be expanded from the existing banked and tested product.
The ongoing clinical trials of our CTX stem Cell Therapy candidates for stroke and critical limb ischaemia utilise a reformulated, cryopreserved variant of the CTX stem cell line, designated CTXcryo. We have generated data demonstrating the equivalence of CTXcryo drug product to the original non-cryopreserved variant. CTXcryo will provide the business with major commercial and competitive advantages in terms of the availability of a genuine off-the-shelf cell-based treatment with a competitive cost of goods and a shelf life enabling shipping to, and storage at, clinical sites on a global basis.
Pre-clinical and clinical results from our technologies and stem cell products have been published extensively in peer-reviewed scientific journals
Human retinal progenitor cells (hRPC)
hRPC are cells that differenitiate into components of the retinia. These cells are used allogeneically and are grown using a patented low-oxygen cell expansion technology licensed from the Schepens Eye Research Institute at Harvard Medical School. Through our collaboration with Schepens we have developed the ability to scale hRPCs using this technology and we have established GMP-compliant hRPC cell bank to provide future drug product.
Exosomes are nanoparticles containing key proteins and micro RNAs. They play a key role in cell-to-cell communication, modulate cellular immunity and promote the activation of regenerative or repair programs in diseased or injured cells. Our CTX cells release large amounts of exosomes when cultured and we have purified and characterised these, testing them at differing concentrations in a range of early in vitro pre-clinical disease models with positive results.