Treatment of SMA type 1 infants using a single-dose AAV9-mediated gene therapy via intrathecal injection of GC101: An open-label, single-arm study

• Xiuwei Ma,
• Lu Zhuang,
• Wenhao Ma,
• Jun Li,
• Xiaodong Wang,
• Zhongqiu Li,
• Xinyang Jiang,
• Yongxia Wang,
• Ying Du,
• Yingqian Zhang,
• Fang He,
• Zhiming Zhu,
• Shaopeng Du,
• Juan Xu,
• Ruijie Gu,
• Yanping Zhang,
• Shan Zhang,
• Ting Li,
• Xiao Yang,
• Sheng Zhang,
• Lina Zhu,
• Qiuping Li,
• Xiaoyan Dong,
• Xiaobing Wu,
• Zhichun Feng,
• Jing Ni

Affiliations

Xiuwei Ma

1 Department of Neurodevelopment, Faculty of Pediatrics, The Seventh Medical Center of Chinese PLA General Hospital, Beijing 100700, China

Lu Zhuang

1 Department of Neurodevelopment, Faculty of Pediatrics, The Seventh Medical Center of Chinese PLA General Hospital, Beijing 100700, China

Wenhao Ma

3 Research and Development Division, Genecradle Therapeutics Inc., Beijing 100176, China

Jun Li

2 National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing 100700, China

Xiaodong Wang

4 Medical Division, Genecradle Therapeutics Inc., Beijing 100176, China

Zhongqiu Li

2 National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing 100700, China

Xinyang Jiang

2 National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing 100700, China

Yongxia Wang

1 Department of Neurodevelopment, Faculty of Pediatrics, The Seventh Medical Center of Chinese PLA General Hospital, Beijing 100700, China

Ying Du

4 Medical Division, Genecradle Therapeutics Inc., Beijing 100176, China

Yingqian Zhang

4 Medical Division, Genecradle Therapeutics Inc., Beijing 100176, China

Fang He

1 Department of Neurodevelopment, Faculty of Pediatrics, The Seventh Medical Center of Chinese PLA General Hospital, Beijing 100700, China

Zhiming Zhu

4 Medical Division, Genecradle Therapeutics Inc., Beijing 100176, China

Shaopeng Du

1 Department of Neurodevelopment, Faculty of Pediatrics, The Seventh Medical Center of Chinese PLA General Hospital, Beijing 100700, China

Juan Xu

5 Department of Pharmacy, Medical Supplies Center of Chinese PLA General Hospital, Beijing 100700, China

Ruijie Gu

1 Department of Neurodevelopment, Faculty of Pediatrics, The Seventh Medical Center of Chinese PLA General Hospital, Beijing 100700, China

Yanping Zhang

2 National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing 100700, China

Shan Zhang

6 Beijing Key Laboratory of Pediatric Organ Failure, Beijing 100700, China

Ting Li

1 Department of Neurodevelopment, Faculty of Pediatrics, The Seventh Medical Center of Chinese PLA General Hospital, Beijing 100700, China

Xiao Yang

2 National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing 100700, China

Sheng Zhang

2 National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing 100700, China

Lina Zhu

2 National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing 100700, China

Qiuping Li

6 Beijing Key Laboratory of Pediatric Organ Failure, Beijing 100700, China

Xiaoyan Dong

4 Medical Division, Genecradle Therapeutics Inc., Beijing 100176, China

Xiaobing Wu

3 Research and Development Division, Genecradle Therapeutics Inc., Beijing 100176, China

Zhichun Feng

1 Department of Neurodevelopment, Faculty of Pediatrics, The Seventh Medical Center of Chinese PLA General Hospital, Beijing 100700, China

Jing Ni