AveXis, Inc. Information

Based in Chicago, Illinois, AveXis is a clinical-stage gene therapy company, dedicated to developing and commercializing novel treatments for patients suffering from rare and life-threatening neurological genetic diseases. Our initial product candidate, AVXS-101, is our proprietary gene therapy product candidate currently in a Phase 1 clinical trial for the treatment of spinal muscular atrophy, or SMA, Type 1, the leading genetic cause of infant mortality. SMA Type 1 is a lethal genetic disorder characterized by motor neuron loss and associated muscle deterioration, resulting in mortality or the need for permanent ventilation support before the age of two for greater than 90% of patients. We are passionately committed to moving gene therapies into the clinical setting for patients and families devastated by rare and orphan neurological genetic diseases. With the support of industry and academic alliances, we're advancing cutting-edge science in order to treat rare and life-threatening genetic diseases—starting with our clinical-stage, proprietary gene therapy candidate, AVXS-101. How to reach us: AveXis, Inc. 2275 Half Day Road, Suite 200 Bannockburn, IL 60015 Email: info@avexis.com Office Phone: 847.572.8280 Toll-free Phone: 844.4.AVEXIS (844.428.3947)