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AveXis Inc.

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AveXis Announces Opening of Enrollment for Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 at Nationwide Children's Hospital

Dallas, TX, May 16, 2014 --( AveXis, Inc., a clinic-ready synthetic biology platform company, today announced that the Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 has now formally begun enrollment at Nationwide Children’s Hospital. This trial will utilize chariSMA, the gene therapy product developed by Dr. Brian Kaspar, of Nationwide Children’s Hospital, and licensed to AveXis for development and commercialization.

“I'm thrilled to see chariSMA translate from the bench to the bedside and am hopeful that we will be able to make a difference for SMA patients,” said Dr. Brian Kaspar.

“We are quite excited about achieving this key milestone and that Nationwide Children’s Hospital can now begin formally recruiting and enrolling candidate subjects for this trial,” said Mindy Du, Director of Scientific and Corporate Affairs at AveXis.

The clinical trial is led by Dr. Jerry Mendell, at The Research Institute at Nationwide Children’s Hospital, having received IND approval and Fast Track designation in September 2013.

For further details on the trial, enrollment criteria, eligibility and contact information please see the complete posting, which can be found at

About Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is an autosomal-recessive genetic disorder characterized by progressive weakness of the lower motor neurons. SMA is caused by a genetic defect in the SMN1 gene which codes SMN, a protein necessary for survival of motor neurons. SMA kills more infants than any other genetic disease in today's world.

About AveXis, Inc.
Based in Dallas, Texas, AveXis is a clinic-ready synthetic biology platform company establishing unique industry alliances to create innovative treatments for people with unmet medical needs. Spinal muscular atrophy (SMA) is the company’s first focus. For more information regarding AveXis, please visit

About Nationwide Children’s Hospital
Ranked in all 10 specialties in “U.S. News & World Report’s 2013-14 Best Children’s Hospitals” and among the Top 10 on Parents magazine’s 2013 “Best Children’s Hospitals” lists, Nationwide Children’s Hospital is one of the nation’s largest not-for-profit freestanding pediatric healthcare networks providing care for infants, children, adolescents and adult patients with congenital disease. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children’s Hospital faculty train the next generation of pediatricians, scientists and pediatric specialists. The Research Institute at Nationwide Children’s Hospital is one of the top 10 National Institutes of Health-funded free-standing pediatric research facilities in the U.S., supporting basic, clinical, translational and health services research at Nationwide Children’s Hospital.
Contact Information
AveXis Inc.
Jillian Bowman

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