AveXis Secures $65 Million in Financing

Company builds investor syndicate comprising leading mutual and biotech funds to advance its spinal muscular atrophy gene therapy program


Chicago, Ill. – AveXis, Inc., a leading gene therapy company developing treatments for rare and life-threatening neurological genetic diseases, today announced the completion of a $65 million Class D common stock financing. The proceeds will be used to advance the company's ongoing spinal muscular atrophy (SMA) clinical program and expand key operational capabilities.

The Class D financing was led by funds and accounts managed by T. Rowe Price Associates, Inc., a new investor in AveXis. Existing investors, including Deerfield Management, Roche Venture Fund and Venrock, also participated in the financing, as did new participants, including Janus Capital Management LLC, Adage Capital Management, L.P., RA Capital Management, QVT Financial LP, Rock Springs Capital Management LP, Foresite Capital Management, LLC, RTW Investments, LLC and Boxer Capital of Tavistock Life Sciences.

"We are pleased to have such a robust syndicate of well-respected investors, led by T. Rowe Price funds and accounts, join with our existing investors in this financing. This points to the broad enthusiasm surrounding our SMA clinical program and the progress we have made to date," said Sean P. Nolan, president and chief executive officer, AveXis. "We are now well-positioned to continue development of our novel gene therapy for patients suffering from SMA Type 1, a rare genetic disease and the most common genetic cause of infant mortality."

AveXis has an ongoing Phase 1 dose-ranging clinical trial utilizing scAAV9.CB.SMN in patients with SMA Type 1. This gene transfer therapy introduces fully-functioning copies of a survival motor neuron (SMN) gene. The copied gene is intended to supplement the body's production of SMN protein using an adeno-associated viral vector that carries the SMN gene. Following this treatment, the company believes that the cell machinery could then continually produce SMN protein from the new copies of the SMN gene.

"SMA is a devastating genetic disease with no approved treatments," said Taymour Tamaddon, portfolio manager, T. Rowe Price Health Sciences Fund. "We believe the AveXis program holds great promise for patients suffering from SMA and we are pleased to support the company."

Cooley LLP served as legal advisor to the company and Jefferies LLC acted as financial advisor to the company for the transaction.

About AveXis, Inc.
AveXis is a clinical-stage gene therapy company developing treatments for rare and life-threatening neurological genetic diseases. The company's initial product candidate, scAAV9.CB.SMN, is in an on-going Phase 1 clinical trial for the treatment of spinal muscular atrophy (SMA) Type 1, 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 that codes SMN, a protein necessary for survival of motor neurons. SMA Type 1 is the most common cause of infant mortality and is the second-most common autosomal recessive genetic disorder. For more information about AveXis, please visit www.avexisbio.com


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