Retinal stem cell data paves way for clinical research

ReNeuron’s hRPC stem cells arrest visual decline in pre-clinical models of retinal degeneration, demonstrating their therapeutic potential.

ReNeuron Group plc, a UK-based stem cell research company, today revealed further key pre-clinical efficacy data with its human retinal progenitor cells (hRPCs). A Phase I/II clinical trial has recently commenced in the US to evaluate the safety, tolerability and preliminary efficacy of the hRPCs in patients with the inherited blindness-causing disease, retinitis pigmentosa (RP).

The study describes work undertaken in conjunction with ReNeuron’s research collaborators at UCL Institute of Ophthalmology, London, and Schepens Eye Research Institute of Massachusetts Eye and Ear, Boston, US and assessed the long-term efficacy and safety of ReNeuron’s hRPCs using established pre-clinical rodent models, finding positive results in these trials.

Preserving and protecting retinal structure

The hRPCs were well tolerated, safe and preserved retinal structure and vision up to six months post-injection in the studies. The cells also survived and integrated into both damaged and normal neural retina without adverse effects.

These findings suggest that, as well as treating later stage retinal disease, the hRPCs can be deployed in less affected regions of the retina at earlier stages of retinal disease or even in normal regions of peripheral retina.

ReNeuron used the above pre-clinical data, as well as earlier, positive pre-clinical data to support the clinical development of its hRPC cell therapy candidate, initially in patients with RP.

Phase I and II clinical trials

The ongoing Phase I/II clinical study is being conducted at Massachusetts Eye and Ear in Boston, a teaching affiliate of Harvard Medical School.

Initial short-term safety and tolerability data from the Phase I part of the study in the first nine patients are expected in early 2017. Longer-term safety data, as well as efficacy read-outs from the Phase II part of the study in a further six patients, are expected in the second half of 2017.