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.
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.
Current Phase: FDA Approved
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