SAPPHIRE–Registration phase study in Canada to establish the efficacy and safety of repeat dosing of autologous endothelial progenitor cells (EPCs) isolated from a patient, transfected with human endothelial nitric oxide synthase (eNOS) and expanded ex-vivo, and then delivered back to the same patient with PAH. The primary therapeutic mechanism is paracrine expression and secretion of eNOS at the human pulmonary vascular interface by long lived patient derived cells. Cells are expanded 2000-fold following retrieval, are optimized to express the natural vasodilating enzyme, eNOS, and cells are stored frozen for monthly administration.
SAPPHIRE is an autologous endothelial cell therapy where the defective gene for nitric oxide (NO), a vasodilator, is added to the patient’s own cells. The patient’s cells carrying the NO gene are given back as simple monthly infusions which improve symptomatic PAH and result in regeneration of blood vessels in the lung.
The SAPPHIRE study is being conducted by an affiliate of United Therapeutics in Canada called Northern Therapeutics, Inc. United Therapeutics has a 49.7 percent voting stake and a 71.8 percent financial stake in Northern Therapeutics. United Therapeutics also has the exclusive right to pursue this technology in the United States if SAPPHIRE is successful.