- "AVXS-101 Phase 1 Gene Therapy Clinical Trial in SMA Type 1: Experience with Preexisting Anti-AAV9 Antibody in the SMA1 Population;" oral presentation on April 24 at 1:00 p.m. during S13: Motor Neuron Diseases: Biomarkers, Outcome Measures and Therapeutics.
- "AVXS-101 Phase 1 Gene Therapy Clinical Trial in SMA Type 1: Correlation between CHOP-INTEND and Motor Milestone Achievements;" oral presentation on April 24 at 1:12 p.m. during S13: Motor Neuron Diseases: Biomarkers, Outcome Measures and Therapeutics.
- "CSF Delivery of AAV9-mediated Gene Therapy for SMA, a Lethal Neuromuscular Disease in Children: A Dose-response Study in Mice and Nonhuman Primates;" poster presentation on April 25 at 5:30 - 7:00 p.m.
About AVXS-101AVXS-101 is a proprietary gene therapy candidate of a one-time treatment for SMA Type 1 and is currently in development for SMA. AVXS-101 is designed to address the monogenic root cause of SMA and prevent further muscle degeneration by addressing the defective and/or loss of the primary SMN1 gene. AVXS-101 also targets motor neurons providing rapid onset of effect, and crosses the blood brain barrier allowing an IV dosing route and effective targeting of both central and systemic features.About AveXis, Inc.AveXis is a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases. The company's initial proprietary gene therapy candidate, AVXS-101, is being evaluated in a Phase 1 clinical trial for the treatment of SMA Type 1. For additional information, please visit www.avexis.com.
Media Inquiries:Lauren BarbieroW2O Group646email@example.comInvestor Inquiries: Jim GoffAveXis, Inc.firstname.lastname@example.org