AveXis Announces Dosing of First Patient in Intermediate Cohort in Gene Transfer Trial

Dallas, TX, December 10, 2014 --(PR.com)-- AveXis, Inc., a biotechnology company focused on using gene therapy to transform the lives of patients with severe genetic and orphan diseases like Spinal Muscular Atrophy (SMA), today announced that the first patient in the intermediate cohort in the Gene Transfer Clinical Trial for SMA Type 1 has been dosed. The world’s first human gene therapy trial for the treatment of infants with SMA (NCT02122952) evaluates chariSMA for safety and efficacy in a 3-cohort design. ChariSMA is a gene therapy product that utilizes AAV9 to deliver the functional SMN gene to SMA patients.

“We are incredibly pleased with the progression of the current trial for the first gene therapy treatment of SMA. The additional cohorts will provide further safety, efficacy, and dosing information that will guide future studies, such as the intrathecal trial we have planned for 2015,” stated John A. Carbona, Chief Executive Officer of AveXis.

The current study is an open-label, single-dose, dose-escalation study, which will enroll nine infants under the age of nine months who have been diagnosed with SMA Type 1. The study is being conducted by Dr. Jerry Mendell at The Research Institute at Nationwide Children’s Hospital. For further details on the trial, enrollment criteria, eligibility, and contact information, please visit www.clinicaltrials.gov/ct2/show/NCT02122952.

To learn more about the Gene Transfer Clinical Trial, other available trials for SMA, and SMA patient advocacy group, please visit www.smastudy.org, a patient-oriented, SMA-focused web resource.

About Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is an autosomal-recessive genetic disorder characterized by lower motor neuron loss and progressive muscle weakness. SMA is caused by a genetic defect in the SMN1 gene which encodes for 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-stage gene therapy 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 7th of only 10 children's hospitals on U.S. News & World Report’s 2014-15 “America’s Best Children’s Hospitals Honor Roll” and among the Top 10 on Parents magazine’s 2013 “Best Children’s Hospitals” list, Nationwide Children’s Hospital is one of the nation’s largest not-for-profit freestanding pediatric healthcare networks providing care for infants, children and adolescents as well as adult patients with congenital disease. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children’s 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. The Research Institute encompasses three research facilities totaling 525,000 square feet dedicated to research. More information is available at NationwideChildrens.org/Research.