AveXis Announces Dosing of First Patient for Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 at Nationwide Children’s Hospital

Dallas, TX, June 26, 2014 --(PR.com)-- AveXis, Inc., a synthetic biology platform company, today announced that the first patient in the Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 has now been enrolled and dosed by Nationwide Children’s Hospital. This trial utilizes chariSMA, the gene therapy product developed by Dr. Brian Kaspar, of Nationwide Children’s Hospital, and licensed to AveXis for development and commercialization. chariSMA utilizes AAV9 to deliver the functional SMN gene to patients. In preclinical studies, AAV9 has been shown to cross the blood-brain-barrier, delivering SMN to motor neurons and significantly improve survival and motor function in an animal model of SMA.

“Over the past few months so many individuals, organizations and foundations have worked tirelessly to bring chariSMA from the bench to the bedside. We are thrilled to be at this inflection point and hopeful that the nonclinical results we have seen will be manifested in the clinic,” said John Carbona, CEO of 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 http://clinicaltrials.gov/ct2/show/NCT02122952.

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 www.avexisinc.com

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.
AveXis Inc.
Jillian Bowman