Novartis Gene Therapies

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Novartis Gene Therapies

AveXis is now Novartis Gene Therapies. Novartis Gene Therapies is dedicated to developing and commercializing gene therapies for patients and families devastated by rare and life-threatening neurological genetic diseases.

AveXis renamed Novartis Gene Therapies, signifying the growing importance of gene therapy to Novartis corporate strategy

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About Novartis Gene Therapies

Since 2013, Novartis Gene Therapies (formerly AveXis) has had one focus: bringing change to those devastated by genetic diseases.

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Understanding Spinal Muscular Atrophy (SMA)

SMA is a rare and devastating genetic disease caused by a lack of a functional survival motor neuron 1 (SMN1) gene, resulting in the rapid and irreversible loss of motor neurons.

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Newborn screening for SMA

In its most severe forms, spinal muscular atrophy can progress rapidly. Early diagnosis through newborn screening can help detect the disease before symptoms appear to aid early treatment intervention.

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Clinical Pipeline
  • 6

    Projects in Novartis Gene Therapies pipeline

  • 162

    Projects in our clinical pipeline

Managed Access Program

The Novartis Gene Therapies "Managed Access Program" makes investigational or unapproved treatments available to eligible patients with serious or life-threatening diseases.

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Gene Therapy Manufacturing

Novartis Gene Therapies has developed a reproducible manufacturing process to address rare genetic disorders.

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    Rare Disorders Patient Community

    Novartis Gene Therapies strives to act as an ally and resource to the communities affected by the conditions we study.

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    Novartis Gene Therapies Grants

    Novartis Gene Therapies may provide funding or other support through research grants and medical education grants.

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    Job Candidates

    Novartis Gene Therapies is committed to developing and commercializing gene therapies for patients and families devastated by rare neurological genetic diseases. Imagine what you could bring to Novartis Gene Therapies.

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    Transparency and Disclosure

    Our vision is to be a trusted leader in changing the practice of medicine. A big part of gaining this trust is being transparent – being open and clearly disclosing what we do, how we work and where we are successful.

Contact Novartis Gene Therapies

Please direct any of your medical inquiries to the Novartis Gene Therapies MedInfo team in the US, Europe, Middle East and Asia, Latin America & Canada, or Asia-Pacific.

Side effects of Novartis medicines can be reported online or to local Novartis Drug Safety Responsible at