Investors + Media

Corporate Profile

AveXis, Inc. 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 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. In addition to developing AVXS-101 to treat SMA Type 1, we also intend to expand the study of gene therapy into SMA Type 2 and two additional rare neurological monogenic disorders: Rett syndrome (RTT) and a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene.

Recent News

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10/03/17
AveXis to Report Top-line Data from the Phase 1 Clinical Trial of AVXS-101 in SMA Type 1 at the International Annual Congress of the World Muscle Society
09/29/17
AveXis Announces Plan to Initiate Pivotal Trial of AVXS-101 in SMA Type 1 Using Product from New GMP Commercial Process
08/10/17
AveXis Reports Second Quarter 2017 Financial and Operating Results

Upcoming Events

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IR CONTACT

Jim Goff
Vice President, Investor Relations and Corporate Communications
Phone: (650) 862-4134
E-mail: jgoff@avexis.com


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