Retinitis pigmentosa is a group of hereditary diseases of the eye that lead to progressive loss of vision 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 hRPC technology has the potential to preserve existing photoreceptors, potentially reducing or halting further deterioration of vision. In addition, these hRPC cells have been shown to mature into functional photoreceptors that are capable of becoming engrafted into the photoreceptor layer.
A Phase I/IIa clinical trial in RP subjects is currently ongoing in the US. This study which is being conducted at two sites – Massachusetts Eye and Ear Infirmary in Boston and Retinal Research Institute in Pheonix – is an open-label, dose escalation study to evaluate the safety, tolerability and preliminary efficacy of a single, sub-retinal injection of the hRPC stem cell therapy candidate in patients with advanced RP.
The primary endpoint of the study is safety, with patients being followed up for 12 months post-treatment, which includes measurements of visual acuity. The Company recently announced an expansion of this study into subjects with less-impaired vision. As a result, top-line data from this trial is now expected in the second half of 2019.
For an article published by the Foundation for Fighting Blindness regarding this trial click here..