Study Summary
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 2 (SMA 2) patients.
Want to learn more about this trial?
Request More InfoInterventions
GC101GENETIC
Self-complementary AAV9 carrying a codon-optimized SMN coding sequence(coSMN1) driven by CMV enhancer and chicken β-actin promoter
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| Peking University， First Hospital, Department of Pediatrics | Beijing | China | |
| Bayi Children's Hospital, Seventh Medical Center, PLA general hospital | Beijing | China | |
| West China Second University Hospital, Sichuan University | Chengdu | China | |
| Children's Hospital of Chongqing Medical University | Chongqing | China | |
| Tongji Medical college of Huazhong University of Science&Technology, Affiliated Children's Hospital | Wuhan | China |