Study Summary
To evaluate the safety and efficacy of intravenous onasemnogene abeparvovec-xioi in pre-symptomatic patients with SMA and 2 or 3 copies SMN2
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onasemnogene abeparvovec-xioiBIOLOGICAL
A non-replicating recombinant AAV9 containing the complimentary deoxyribonucleic acid (cDNA) of the human SMN gene under the control of the cytomegalovirus (CMV) enhancer/chicken-β-actin-hybrid promoter (CB). The AAV inverted terminal repeat (ITR) has been modified to promote intramolecular annealing of the transgene, thus forming a double-stranded transgene ready for transcription.
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| David Geffen School of Medicine at UCLA | Los Angeles | California | United States |
| Children's Hospital Colorado | Aurora | Colorado | United States |
| Nemours Children's Hospital | Orlando | Florida | United States |
| Massachusetts General Hospital | Boston | Massachusetts | United States |
| Helen DeVos Children's Hospital | Grand Rapids | Michigan | United States |
| St. Louis Children's Hospital | St Louis | Missouri | United States |
| Columbia University Medical Center | New York | New York | United States |
| Nationwide Children's Hospital | Columbus | Ohio | United States |
| Clinic for Special Children | Strasburg | Pennsylvania | United States |
| Children's Medical Center Dallas | Dallas | Texas | United States |
| University Hospital and UW Health Clinics | Madison | Wisconsin | United States |
| Sydney Children's Hospital | Randwick | New South Wales | Australia |
| Centre Hospitalier Régional Hôpital La Citadelle | Liège | Belgium | |
| Canada Childrens Hospital of Eastern Ontario | Ottawa | Ontario | Canada |
| Tokyo Women's Medical | Tokyo | Japan | |
| Great Ormond Street Hospital for Children NHS Foundation Trust | London | United Kingdom |