Press Release

AveXis Closes its Series C Financing Round with Deerfield Management and Roche Venture Fund

Dallas – January 6, 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 it has completed a $10 million financing round led by Deerfield Management and Roche Venture Fund. In conjunction with this financing round, Jonathan Leff of Deerfield and Carole Nuechterlein of Roche Venture Fund will join the Company’s Board of Directors.

Proceeds from the financing will be used to further the development of the Company’s gene therapy product, chariSMATM, including additional support for the current Phase I clinical trial in SMA Type 1 infants. The Research Institute at Nationwide Children’s Hospital received IND approval and Fast Track designation in September 2013. The trial is currently open for enrollment and recruiting candidates (NCT02122952).

“We are delighted to welcome Deerfield and Roche as investors, and Jonathan and Carole to our Board, at an exciting time for AveXis,” said John A. Carbona, Chief Executive Officer at AveXis. “We have initiated the first-ever clinical trial with a gene replacement therapy for SMA, and are now in a position to further advance the product in development as rapidly as possible.”

Mr. Leff is a Partner of Deerfield Management and Chairman of the Deerfield Institute. Prior to joining Deerfield, Mr. Leff was with Warburg Pincus where he led the firm’s investment efforts in biotechnology and pharmaceuticals. In addition to his experience as a director of many public and private biotechnology companies, Mr. Leff serves on the Boards of the Spinal Muscular Atrophy Foundation, the National Venture Capital Association (NVCA), the Biotechnology Industry Organization, Friends of Cancer Research, and the Board of Advisors of Columbia University Medical Center.

“We believe the chariSMATM program holds great promise for patients and families suffering with SMA. The initiation of clinical trials with the first gene replacement therapy for SMA is a significant accomplishment, and we are pleased to have the opportunity to help AveXis accelerate the development of treatments for SMA and other devastating genetic disorders,” said Mr. Leff.

Ms. Nuechterlein has headed the Head of Roche Venture Fund for the past 13 years. Ms. Nuechterlein came to Roche Venture Fund from SangStat where she served as General Counsel. Ms. Nuechterlein currently serves on the board of Lysosomal Therapeutics Inc. and led RVF’s investments in Alios, Ambit, BioFire Diagnostics, Conatus, Envoy, Nereus, and Pharmasset.

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 Deerfield Management
Deerfield is an investment management firm committed to advancing healthcare through investment, information and philanthropy. For more information, please visit www.deerfield.com

About the Roche Venture Fund
The Roche Venture Fund invests in early stage biotech and diagnostics companies to develop commercially successful innovative life science companies. Based in Basel, Switzerland with an office in South San Francisco, the Roche Venture Fund invests globally with a portfolio of over 35 companies in 10 countries. For more information, please visit  www.venturefund.roche.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.


AveXis Contact:
John A. Carbona, Chief Executive Officer
972-725-7797 ext: 201 or jc@avexisinc.com


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