Novartis plans to acquire the neurological gene therapy firm AveXis for $8.7 billion. The purchase will bolster the Swiss giant’s standing as a big pharma leader in the emerging gene therapy field.
AveXis’s leading drug candidate is its gene therapy to treat infants with spinal muscular atrophy type 1 (SMA1), a devastating genetic disease, sometimes called “floppy baby syndrome,” that causes weak muscles and difficulty breathing. Only 8% of afflicted infants survive to 20 months of age. Those that do rely on ventilation support and will never walk.