Drug intelligence / Profile preview

ceNOS gene therapy

Development stage
Preclinical
Lead developer
Johnson & Johnson
Modality
Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intravascular, Inhalation
01

Overview

ceNOS gene therapy is an adenoviral-based gene therapy platform developed through a licensing agreement between Crucell and BIOA&D. The therapy utilizes an adenoviral vector (such as AdCMVceNOS) to deliver the gene encoding human constitutive endothelial nitric oxide synthase (ceNOS/eNOS) into vascular tissues. By restoring or enhancing the production of nitric oxide (NO), the therapy aims to counteract endothelial dysfunction and impaired NO bioavailability. Primary indications investigated in preclinical research include pulmonary hypertension, where it reduces hypoxic pulmonary vasoconstriction, and the prevention of restenosis following balloon angioplasty, where it inhibits vascular smooth muscle cell proliferation and neointima formation. The platform has demonstrated efficacy in rat and porcine models of arterial injury and pulmonary vascular disease.

Other names
eNOS gene therapyendothelial nitric oxide synthase gene therapyconstitutive endothelial nitric oxide synthase gene therapyAd-eNOS
02

Targets

NOS3 (eNOS)

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