Onasemnogene Abeparvovec-Xioi‎

Disease:  Spinal Muscular Atrophy

Indication:  For the treatment of pediatric patients less than 2 years of age with SMA with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

Company:  AveXis

Private Company

Notable Transactions:   Based on the NAV AAV9 vector that was developed by RegenXBio. AveXis was acquired by Novartis $8.7B.

Methods:  Utilizes NAV AAV9 to deliver functional copies of the SMN1 gene to neurons.

Link to the clinical trial:  NCT03306277

Link to company R&D pipeline:  https://avexis.com/research-and-development.html

Current Phase:  FDA Approved

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