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 1 (SMA 1) patients.
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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 | |
| Shengjing Hospital of China Medical University | Shenyang | China | |
| Children's Hospital of Soochow University | Suzhou | China |