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AveXis Inc.

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AveXis Closes a Round of Financing from PBM Capital to Advance Its SMA Gene Therapy Program

Dallas, TX, April 23, 2014 --( AveXis, Inc., a clinic-ready synthetic biology platform company, today announced it has completed a financing round led by PBM Capital Group. This round of financing will help support the company’s gene therapy program targeting spinal muscular atrophy (SMA). AveXis’ gene therapy product, chariSMA, is a unique approach to the disease which affects more infants than any other genetic disease in the world today. In conjunction to the funding, PBM will join AveXis’ Board of Directors.

“Securing this capital not only provides AveXis with additional resources to advance its SMA gene therapy program both commercially and clinically but also aligns well with the company’s strategy of integrating entrepreneurship and philanthropy,” said Mindy Du, Director of Scientific and Corporate Affairs at AveXis.

This round of financing will also help fund the Phase 1 clinical trial for the chariSMA gene therapy product in SMA Type 1 patients. The clinical trial, led by Dr. Jerry Mendell, is slated to begin in the second quarter of 2014 at The Research Institute at Nationwide Children’s Hospital, having received IND approval and Fast Track designation in September 2013.

“AveXis and Nationwide Children’s are continually striving to aggressively advance chariSMA to market for patients afflicted with SMA. Our focused efforts have resulted in the signing of an exclusive license to ReGenX’s NAV rAAV9 vector for SMA and the initiation of a clinical trial within the coming months,” said Minna Du, Director of Clinical Operations and Research.

“This trial represents the best of 'bench-to-bedside' translational research. Dr. Mendell and his team have designed a trial that will test the safety of the therapy and be able to show early measures of efficacy,” said Allan Kaspar, Chief Scientific Officer of AveXis.

With the completion of this round of funding, AveXis is also proud to announce their new national headquarters at One Energy Square in Dallas, Texas.

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 PBM Capital Group
PBM Capital Group, LLC is a private investment firm focused on healthcare and life science investments. The firm was founded and is led by Paul B. Manning, an entrepreneur who most recently sold an infant formula and nutrition business to Perrigo Company (NYSE: PRGO, TASE) in 2010. PBM Capital invests across all stages of corporate development: venture capital, leveraged buyouts, growth capital, distressed/turnaround, and public equity value investing. Portfolio companies also include Revive Pharmaceuticals, Enzymatics, Triangle Research Labs, Firefly Bioworks, Genometry 9, and QC Labs. Visit for more information.

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.
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AveXis Inc.
Jillian Bowman

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