eNOS-transfected autologous endothelial progenitor cells (Northern Therapeutics)

This is an experimental autologous cell-based gene therapy developed by Northern Therapeutics for the treatment of severe, refractory pulmonary arterial hypertension (PAH). The therapy involves harvesting a patient's own endothelial progenitor cells (EPCs), which are then transiently transfected ex vivo with the human endothelial nitric oxide synthase (eNOS or NOS3) gene. These modified cells are delivered via a pulmonary artery catheter into the right atrium. Once in the pulmonary circulation, the EPCs are intended to home to damaged microvasculature and express eNOS, which catalyzes the production of nitric oxide (NO). NO serves as a potent vasodilator and inhibitor of vascular remodeling. This dual approach aims to both provide immediate hemodynamic relief and promote long-term vascular repair and regeneration. The approach was evaluated in the Phase I PHACeT trial.

Other names

eNOS-transfected EPCsheNOS-EPCsautologous progenitor cell-based gene therapy of heNOSautologous EPC-eNOS gene therapy