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AveXis and BioLife Announce The Research Institute at Nationwide Children’s Hospital Received Fast Track Status for Spinal Muscular Atrophy Treatment

Columbus, OH - 10/17/2013

AveXis and BioLife, synthetic biology platform companies, today announced that The Research Institute at Nationwide Children’s Hospital received Fast Track designation from the U.S. Food and Drug Administration for its scAAV9.CB.SMN gene therapy product for the treatment of spinal muscular atrophy (SMA). This new gene therapy product created by scientists at The Research Institute was granted Fast Track status after demonstrating preliminary effectiveness in mouse models of SMA, potentially addressing this unmet medical need.

"Fast track," an important hastened phase in the nation's drug review and approval process, signifies that the FDA can expedite the review and development of the scAAV9.CB.SMN gene therapy product which, in preclinical work, has shown to slow the progression of SMA symptoms.

SMA is the most common genetic cause of infant death. SMA is an autosomal recessive disease caused by a genetic defect in the SMN1 gene that encodes for the SMN protein. SMA manifests in various degrees of severity which all have in common general muscle wasting and mobility impairment.

The scAAV9.CB.SMN gene therapy product currently being developed by The Research Institute, received an IND approval in September 2013 to initiate Phase I clinical testing in  SMA Type 1patients.The clinical trial is sponsored by The Sophia’s Cure Foundation and AveXis/BioLife.

“Today’s notice of Fast Track designation is welcomed news. This demonstrates to the families afflicted with SMA, the FDA’s regulatory priority for addressing this orphan disease,” said Chief Executive Officer John A. Carbona.

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 Nationwide Children’s Hospital
Ranked in all 10 specialties in “U.S. News & World Report’s 2013-2014 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.

About AveXis, Inc.
Based in Dallas, Texas, AveXis is a 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.

Corporate Contact:

John A. Carbona, Chief Executive Officer
972-331-1905 or

Media Contact:

Jillian Bowman, Administrative Specialist
972-331-9626 or

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Nationwide Children's Hospital
700 Children's Drive Columbus, Ohio 43205 614.722.2000