AveXis is a biotechnology company that is 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, the company is advancing cutting-edge science in order to treat rare and life-threatening genetic diseases—starting with their clinical-stage, proprietary gene therapy candidate, AVXS‑101. AVXS-101 has generated highly promising clinical data in patients with Spinal Muscular Atrophy Type I, the most devastating form of SMA.