Year 3

Over the past year, significant progress has been made in advancing our therapy for retinitis pigmentosa (RP). We have initiated our clinical trials at the Gavin Herbert Eye Institute at UCI and Retina Vitreous Associates (RVA) to test the safety of a single, intravitreal injection of human retinal progenitor cells (jCell) in patients with advanced RP to validate that the procedure is safe. As the therapy has been designed to preserve vision by intervening in the disease at a time when host photoreceptors can be protected and potentially reactivated, the trial is also designed to assess, as a secondary outcome measure, the effect of treatment on ocular function. Eight patients in our first cohort and one patient in our second cohort have been injected at two different dose levels: 0.5 million and 1.0 million cells. Our DSMB reviewed our safety data after the fourth and eight patient and had no safety concerns. We look forward to completing enrollment in the first half of 2016. Following enrollment, the patients are followed for a 12 month period to determine if the therapy is safe and look for early indications of beneficial effects on vision.