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“AVXS‐101 Phase 1 Gene Therapy Clinical Trial in SMA Type 1: Event-free Survival and Achievement of Developmental Milestones” will be presented by
"We are eager to present these data from the first ever clinical trial of gene therapy in spinal muscular atrophy," said
A total of five preclinical and clinical abstracts will be presented during the meeting, underscoring the company’s leadership in pioneering gene therapy to treat life-threatening neurological diseases. In addition to the presentations highlighted above, these include:
- “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 24at 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 24at 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 25at 5:30 – 7:00 p.m.
All abstracts can be accessed through the AAN website at www.aan.com.
SMA is a severe neuromuscular disease characterized by the loss of motor neurons leading to progressive muscle weakness and paralysis. SMA is caused by a genetic defect in the SMN1 gene that codes SMN, a protein necessary for survival of motor neurons. The incidence of SMA is approximately one in 10,000 live births.
The most severe form of SMA is Type 1, a lethal genetic disorder characterized by motor neuron loss and associated muscle deterioration, which results in mortality or the need for permanent ventilation support before the age of two for greater than 90 percent of patients. SMA Type 1 is the leading genetic cause of infant mortality.
AVXS-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.
Lauren Barbiero W2O Group646-564-2156 firstname.lastname@example.org Investor Inquiries: Jim Goff AveXis, Inc.650-862-4134 email@example.com