eNOS

eNOS (Endothelial Nitric Oxide Synthase) is an adeno-associated virus (AAV)-based gene therapy developed by Target Heart (a subsidiary of Asklepios BioPharmaceutical, or AskBio) for the treatment of cardiovascular diseases, specifically heart failure and endothelial dysfunction. The therapy, also referred to as TH-001, utilizes an AAV vector to deliver the NOS3 gene directly to the cardiac or vascular tissues. The goal is to restore or enhance the production of nitric oxide (NO), a critical signaling molecule that promotes vasodilation, inhibits platelet aggregation, and reduces inflammation and oxidative stress within the vasculature. By increasing eNOS expression, the therapy aims to improve myocardial perfusion and overall cardiac function. The program was identified in AskBio's pipeline following its acquisition of Target Heart, Inc., and has been evaluated in Phase 1 clinical development.

Other names

eDINAAV-eNOSNOS3 gene therapyNOS-3 gene therapyNOS 3 gene therapyEndothelial Nitric Oxide Synthase gene therapy