Drug intelligence / Profile preview

autologous eNOS-transfected endothelial progenitor cells

Development stage
Unknown
Lead developer
Northern Therapeutics
Modality
Gene Therapies, Stem Cell Therapies → Cell Therapies
Administration
Intravenous
01

Overview

This experimental therapy is a combination of cell and gene therapy developed by Northern Therapeutics for the treatment of severe, refractory pulmonary arterial hypertension (PAH). The approach involves harvesting autologous endothelial progenitor cells (EPCs) from the patient, which are then cultured and transiently transfected *in vitro* with a plasmid encoding human endothelial nitric oxide synthase (eNOS). These modified cells are re-infused into the patient's right atrium via a pulmonary artery catheter. The therapy aims to repair damaged pulmonary vasculature and promote regeneration by delivering eNOS directly to the lungs, thereby increasing local nitric oxide production to induce vasodilation and improve vascular health.

Other names
eNOS-transfected EPCsautologous progenitor cell-based gene therapy delivery of heNOSPHACeT therapy
02

Targets

NOS3 (eNOS)

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