GC801-enabled AAV gene therapy in a neonate with Pompe disease
Jia Chen, Wenhao Ma, 漆洪波, Xiuwei Ma, Yanmei He, Liu He, Yan Ke, Lu Zhuang, Xinyang Jiang, Jianhua Wang, Juan Xu, Yan Xia, Jianfang Zhou, Yue Wang, Xiaoyan Dong, , Zhichun Feng
This paper describes the use of GC801 to reduce anti-AAV9 antibodies in a neonate with Pompe disease, enabling successful AAV9 gene therapy.
medRxiv · Sep 29, 2026 · PreprintGenetic & Genomic Medicine