Abstract
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. In November, a study showed that all 15 infants treated with AveXis’s gene therapy for SMA1 were alive after 20 months (N. Engl. J. Med. 2017, DOI: 10.1056/nejmoa1706198). “Those kids were dying, and the outcomes look really good at this point,” Mark A. Kay, a director of the human gene therapy program at Stanford University School of Medicine, told C&EN in November when the study was released. Gene therapy enthusiasm grew even more in December
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