AAV-hPGIS

AAV-hPGIS is a **gene therapy** that uses an adeno-associated virus (AAV) vector to deliver the human prostaglandin I2 synthase (hPGIS) gene into host tissue, with the aim to treat **pulmonary arterial hypertension (PAH)**. The therapy leverages the vasodilatory, antiplatelet, and anti-proliferative effects of prostaglandin I2 (prostacyclin), but instead of requiring continuous infusion of the prostacyclin molecule, it enables sustained endogenous production of prostacyclin via muscle-targeted delivery of the hPGIS gene. Studies have demonstrated efficacy in animal models, with both AAV serotype 1 and 2 proving effective at reducing key disease metrics, such as right ventricular systolic pressure, right ventricular hypertrophy, and vascular remodeling. Delivery is typically via intramuscular injection[1][2][3].

Other names

adeno-associated virus encoding human prostaglandin I2 synthaseAAV-hPGIS gene therapy