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AveXis Announces the Formation of its European Subsidiary, AveXis EU, Ltd.

Dallas – February 25, 2015 – 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 the formation of its EU-based wholly-owned subsidiary, AveXis EU, Ltd. The subsidiary will allow AveXis to move forward with its plans to open clinical trials in the European Union and leverage European Medicines Agency’s programs, such as Orphan Medicinal Product Designation and the Micro-, Small-, and Medium-Sized-Enterprise (SME) program, to advance development.

Similar to the FDA Orphan Drug Designation, the EMA Orphan Designation grants eligible companies several benefits, such as reduced fees for protocol assistance and scientific advice and market exclusivity for 10 years once the drug is granted approval. The EMA, recognizing the need to encourage drug development by small biotech companies, also established the SME program in late 2005 which offers administrative and procedural assistance, among other incentives.

“We are excited to begin planning for the expansion of AveXis’ presence in the European Union. Establishing an EU-based subsidiary is our first step in opening communications between the regulatory agencies that govern the conduct of clinical trials in Europe and AveXis,” commented John A. Carbona, Chief Executive Officer of AveXis.

AveXis is currently sponsoring a US-based Phase 1 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 at The Research Institute at Nationwide Children’s Hospital. The trial is currently open for enrollment and recruiting candidates. For further details on the trial, enrollment criteria, eligibility, and contact information, please visit http://clinicaltrials.gov/ct2/show/NCT02122952.

AveXis has also continued its efforts to grow awareness of the ongoing and upcoming clinical trials with the Contact All Neurologists (C.A.N.) ProgramTM. The C.A.N. ProgramTM has reached over 30 neurologists nationwide, informing them of clinical trials available to infants recently diagnosed with SMA Type 1. For neurologists, pediatricians, or physicians interested in participating in the AveXis C.A.N. ProgramTM, please contact CAN@avexisinc.com.

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 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-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.avexis.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. ,/p>

AveXis Contact:
John A. Carbona, Chief Executive Officer
972-725-7797 or jc@avexisinc.com


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