autologous heNOS-engineered mononuclear cells

autologous heNOS-engineered mononuclear cells (commercially known as **Aurora-GT**) is an investigational cell-based gene therapy developed by **Northern Therapeutics** in collaboration with **United Therapeutics** and **MaxCyte**. The therapy consists of a patient's own (autologous) endothelial progenitor cells (EPCs) that are harvested via apheresis and genetically engineered *ex vivo* using MaxCyte's non-viral electroporation technology to transiently express human endothelial nitric oxide synthase (heNOS, encoded by the *NOS3* gene). Once delivered back to the patient via the pulmonary circulation, these engineered cells are designed to home to damaged lung vasculature, restore endothelial function, promote vasodilation, and attenuate or reverse vascular remodeling by overexpressing eNOS and boosting local nitric oxide (NO) production. The therapy has been evaluated in the Phase I PHACeT trial and the Phase II/III SAPPHIRE trial for the treatment of severe pulmonary arterial hypertension (PAH), including idiopathic, familial, and anorexigen-induced forms. However, the SAPPHIRE trial was terminated in early 2025 due to lower-than-anticipated enrollment.

Brand names

Aurora-GT

Other names

eNOS-transfected EPCseNOS-enhanced EPCsautologous endothelial progenitor cells transfected with human endothelial nitric oxide synthasenitric oxide synthase gene therapynitric oxide synthase therapy