Year 2

Over the past year our laboratory has made significant progress to advance our therapy for retinitis pigmentosa (RP). We are pleased to have finally reached the stage that our human retinal progenitor stem cells will soon be tested in the clinic. We are now at the midway point in our CIRM Disease Team Therapy Development Award and our progress remains on schedule. Thus far, our manufactured cells have undergone the full array of tests in animal models to ensure that they meet all safety requirements and that they are therapeutically potent. Results have been positive, showing that allogeneic retinal progenitor cells survival transplantation to the back of the eye with no immunosuppressive agents required (except when testing across species). Additionally, we have obtained evidence in animal models of graft-associated benefits, both at the anatomical and functional levels. All of these data have been compiled, critically reviewed, and submitted to the FDA to seek approval for early stage clinical trials, thereby achieving all Year 2 Milestones for the project. As part of the approval process to commence trials, we have demonstrated the quality and consistency of the cells to be administered to patients. Each batch of manufactured cells has been shown to be free of microbial contamination and chromosomal defects and has also shown to be stable over time, ensuring suitability for use. The primary purpose of the proposed clinical trial will be to test the safety of a single intravitreal injection of human retinal progenitor cells (jCell) in patients with advanced RP. 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. As a secondary outcome measure, the trial will also measure the effect of our treatment on ocular function. There will be two different dose levels of cells assessed in each of two groups of patients.