22
Participants
Start Date
October 24, 2017
Primary Completion Date
November 12, 2019
Study Completion Date
November 12, 2019
Onasemnogene Abeparvovec-xioi
Non-replicating recombinant adeno-associated virus serotype 9 (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.
Columbia University, New York
Children's Hospital of Philadelphia, Philadelphia
Johns Hopkins Pediatric Neurology, Baltimore
Duke University, Durham
Nemours Children's Hospital, Orlando
Nationwide Children's Hospital, Columbus
University of Wisconsin (Madison), Madison
Ann and Robert H Lurie Children's Hospital, Chicago
Washington Unviersity School of Medicine, St Louis
University of Texas Southwestern Medical Center, Dallas
Children's Hospital Colorado, Aurora
University of Utah, Salt Lake City
David Geffen School of Medicine at UCLA, Los Angeles
Stanford University, Stanford
Oregon Health and Science University, Portland
Boston Children's Hospital, Boston
Lead Sponsor
Novartis Gene Therapies
INDUSTRY