We want to ensure that physicians, researchers, and other healthcare professionals have access to complete, up to date and balanced scientific information regarding gene therapy research and our investigational therapy.

As a company, we are dedicated to developing novel treatments for patients suffering from rare and life-threatening neurological genetic diseases. Our lead product candidate, AVXS-101, is our proprietary gene therapy currently in the pivotal stage of development for the treatment of spinal muscular atrophy (SMA) Type 1 and in a Phase 1 clinical trial for the treatment of SMA Type 2. The U.S. Food and Drug Administration (FDA) has granted AVXS-101 Orphan Drug Designation for the treatment of all types of SMA and Breakthrough Therapy Designation, as well as Fast Track Designation, for the treatment of SMA Type 1. The European Medicines Agency (EMA) also granted AveXis access into its PRIority MEdicines (PRIME) program for AVXS-101 for the treatment of SMA Type 1. The PRIME application was based on data from both preclinical evaluations and the Phase 1 clinical trial of AVXS-101. In addition to developing AVXS-101 to treat SMA Type 1 and Type 2, we also plan to develop other novel treatments for rare neurological diseases, including Rett syndrome (RTT) and a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene.

Please direct any of your medical inquiries regarding gene therapy or AVXS-101 to

You can also learn more by visiting our Research and Development section.