AveXis announces Jonathan Leff and Carole Nuechterlein to its Board after completion $10m financing round

– USA, TX – 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 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 Deerfield Management

Deerfield is an investment management firm committed to advancing healthcare through investment, information and philanthropy.

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.

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