AveXis Announces the Completion of Dosing of the Low Dose Cohort in US Clinical Trial for Spinal Muscular Atrophy

Dallas, TX, October 08, 2014 --(PR.com)-- AveXis, Inc., a clinic-stage gene therapy company, today announced the completion of dosing in the low dose cohort in the world's first human gene therapy trial for the treatment of infants with spinal muscular atrophy (SMA). The trial (NCT02122952) opened for enrollment in April 2014.

“History in gene transfer therapy was made on May 13th, when an infant was successfully injected with about 400 trillion viral particles containing the SMN transgene without any complications to date. That historical moment was followed by the dosing of two additional infants in the low dose cohort,” commented John A. Carbona, Chief Executive Officer of AveXis.

AveXis also announced ongoing plans to modify the current trial design through the addition of a third cohort at a mid-range dose. This marks a departure from the originally-designed 2-cohort trial. “We are also on track to start our intrathecal trial in the first half of 2015 and very serious thought is being given to opening a trial to treat Type 2 patients in 2015. Right now, we are looking at patient recruitment as a gating issue,” said Allan Kaspar, Chief Scientific Officer of AveXis.

In addition, AveXis is announcing the start of its C.A.N. Program. This new initiative is designed to Contact All Neurologists in an effort to grow awareness of the ongoing and upcoming clinical trials in SMA, in addition to informing neurologists how they can help shape the future of patient recruitment in SMA trials. AveXis will also be reaching out to foundations and patient advocacy groups, both domestically and internationally, to increase the overall awareness of clinical trials.

For further details on the trial, enrollment criteria, eligibility and contact information please see the complete posting, which can be found at http://clinicaltrials.gov/ct2/show/NCT02122952.

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 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
AveXis Inc.
Jillian Bowman