This company has developed a cell-based therapy (jCell) to preserve and restore vision in people with retinitis pigmentosa (RP), although the therapy is potentially applicable to other blinding diseases, as the treatment is nonsurgical and minimally invasive. jCyte is fully funded through its Phase IIb trial through the California Initiative in Regenerative Medicine (CIRN), and the company’s platform was granted breakthrough designation in 2017.
Results from a Phase I/IIa trial (n=28) found jCell to be safe and well tolerated; the Phase IIb trial started during Q3 2017 and is actively enrolling. jCell has “extreme efficacy” in RP, said David Boyer, MD, one of the company’s investigators. jCell is human retinal progenitor cells that are not pluripotent; the mechanism of action is neurotrophic (not regenerative or reparative). “jCyte is the only cell therapy program using intravitreal injection of an ocular cell type versus subretinal,” Dr. Boyer said. Waterfall plots showed at six months the majority of patients improved with treatment, and efficacy was between 5 and 10 letters in the 1 million and 2 million cell arms; it trended slightly higher to 10–15 letters in the 3 million cell arm, a trend that continued into 12 months.