Retinitis pigmentosa is 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.
Pre-clinical studies carried out in disease models by the our 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.
The Phase I/II clinical trial in RP patients is now open for enrolment and, importantly, marks the initiation of clinical trial activity in the US with our therapeutic programmes. The study is being conducted at Massachusetts Eye and Ear Infirmary in Boston, a world-renowned clinical centre for the treatment of retinal diseases. 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 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 in the Phase I element of this study in 9 patients is ongoing and we recently announced our intention to expand the Phase II element of this ongoing clinical trial from 6 to 20 patients to provide a richer data set from which to embark on a subsequent Phase II/III pivotal study.
For an article published by the Foundation for Fighting Blindness regarding this trial click here..