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

Projects in our gene therapy pipeline


Projects in our clinical pipeline

Novartis pipeline
Mum holding newborn's feet

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.


Novartis Gene Therapies request

Novartis Gene Therapies wants to ensure that physicians, researchers, and other healthcare professionals have access to complete, up-to-date, and balanced scientific information regarding gene therapy treatment, research and investigational therapies.

Contact Novartis Gene Therapies

Please direct any of your medical inquiries to the Novartis Gene Therapies Medical Information in the US (Healthcare Professionals, Consumers), Europe, Middle East and AfricaLatin America and Canada, or Asia-Pacific and Australia. Visit the Novartis Gene Therapies Medical Information website for additional information.

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

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.