Our human retinal progenitor cell (hRPC) programme for blindness-causing diseases of the retina is partnered with the Schepens Eye Research Institute Massachusetts Eye and Ear, an affiliate of Harvard Medical School in Boston, USA and a world-renowned clinical centre for the treatment of retinal diseases. This programme is initially focused on retinitis pigmentosa (RP), a group of hereditary diseases of the eye that lead to progressive loss of sight due to cells in the retina becoming damaged and eventually dying.
We are using proprietary human retinal progenitor cells as the basis of our programme. Pre-clinical studies carried out in disease models by the Company’s academic collaborators have demonstrated that, when transplanted into the retina, our retinal progenitor cell technology has the potential to preserve existing photoreceptors, potentially reducing or halting further deterioration of vision. In addition, the progenitor cells have been shown to mature into functional photoreceptors that engraft into the photoreceptor layer, bringing the possibility of restored vision. Our hRPC stem cell therapy candidate benefits from Orphan Drug Designation in both Europe and the US. We have worked with world-leading collaborators and academic institutions in the retinal disease field to successfully take our retinitis pigmentosa programme through pre-clinical development and have received regulatory approval from the US Food and Drug Administration (FDA) to commence a Phase I/II clinical trial in the US with our hRPC therapy candidate for RP. This study marks the initiation of clinical trial activity in the US with our therapeutic programme.
The study is being conducted at Massachusetts Eye and Ear Infirmary in Boston. The trial design is an open-label, dose escalation study to evaluate the safety, tolerability and preliminary efficacy of the hRPC stem cell therapy candidate in up to 15 patients with advanced RP. The method of administration of the hRPCs will be a single sub-retinal injection. The primary endpoint of the study is safety, with patients being followed up for 12 months post-treatment with monitoring including measurements of visual acuity. Recruitment and treatment of the first and second dose cohorts has completed.
Please click here to access a webcast of the June 2016 Key Opinion Leader event on Retinitis Pigmentosa.