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AveXis licenses REGENXBIO's NAV AAV9 vector for rare CNS diseases

Executive Summary

In a new deal that builds on their 2014 agreement, AveXis Inc. licensed exclusive worldwide rights to REGENXBIO Inc.'s adeno-associated virus vector NAV AAV9 to develop and commercialize gene therapies for the rare neurological disorders Rett syndrome (RTT; caused by an X-linked dominant mutation in the methyl CpG binding protein 2 (MECP2) gene) and amyotrophic lateral sclerosis (ALS; caused by mutations in the superoxide dismutase 1 (SOD1) gene).
Deal Industry
  • Pharmaceuticals
  • Pharmaceuticals
    • Drug Delivery
      • Macromolecule
  • Biotechnology
    • Gene Therapy, Cell Therapy
    • Large Molecule
Deal Status
  • Final
Deal Type
  • Alliance
    • Includes Contract
    • Includes Royalty or Profit Split Information
    • Intra-Biotech Deal
    • R&D and Marketing (Licensing)

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