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 that is in development.

As a company, we are dedicated to developing 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 development for the treatment of spinal muscular atrophy, or SMA, Type 1, the leading genetic cause of infant mortality. The U.S. Food and Drug Administration, or 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 ongoing Phase 1 clinical trial of AVXS-101. In addition to developing AVXS-101 to treat SMA Type 1, we plan to develop AVXS-101 to treat additional SMA types and develop other novel treatments for rare neurological genetic diseases.

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.