pariglasgene brecaparvovec

**Pariglasgene brecaparvovec** is a liver-directed, one-time adeno-associated virus serotype 8 gene therapy for glycogen storage disease type Ia. It uses an AAV8 vector to deliver a functional copy of the **G6PC** gene, enabling hepatic expression of glucose-6-phosphatase catalytic activity under the native promoter and thereby supporting endogenous glucose release and physiologic glycemic control. It was developed as DTX401 and is marketed in the United States as GENGLYCOS for patients aged 8 years and older with glycogen storage disease type Ia, as an adjunct to nutritional management to reduce daily cornstarch intake. ([ultragenyx.com](https://www.ultragenyx.com/wp-content/uploads/2026/02/401GSDIA02_Lay_Summary_EN.pdf))

Brand names

GENGLYCOS

Other names

AAV8G6PCAAV-8G6PCAAV 8G6PCpariglasgene brecaparvovec-opnr