Company Profile
AveXis Inc.
Company Overview
Our biotech company 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, 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.
Our proprietary gene therapy candidate, AVXS-101, has been granted Orphan Drug Designation for the treatment of all types of spinal muscular atrophy (SMA) and Breakthrough Therapy Designation, as well as Fast Track Designation for the treatment of SMA Type 1—one of the most life-threatening neurological genetic disorders.