gene therapy – it's in our DNA

At AveXis, we are always looking for talented and dedicated people to join our team. Based in Dallas, Texas, AveXis is a clinic-stage gene therapy platform company establishing unique industry and academic alliances to create innovative treatments for people with unmet medical needs.

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Dallas – October 6, 2014 – AveXis Inc., a clinic-stage gene therapy company, today announced the completion of dosing in the low dose cohort in the world’s first human gene therapy trial for the treatment of infants with spinal muscular atrophy (SMA). The trial (NCT02122952) opened for enrollment in April 2014.

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The Phase 1 gene transfer clinical trial using chariSMA™ in SMA Type 1 infants at Nationwide Children’s Hospital is now open for recruitment. For more information, please visit clinicaltrials.gov (NCT02122952). Nationwide Children’s Hospital received IND approval and Fast Track designation from the FDA in…

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AveXis is dedicated to supporting families who are affected by Spinal Muscular Atrophy. We are proud sponsors of organizations like Cure SMA, The Sophia’s Cure Foundation, and Avery’s Race who are trying to spread awareness and support to communities so that we can find a treatment for SMA.

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