BioCentury
ARTICLE | Company News

Breakthrough designation for SMA therapy boosts AveXis

July 20, 2016 7:00 AM UTC

Gene therapy company AveXis Inc. (NASDAQ:AVXS) gained $4.64 (13%) to $40.65 on Wednesday after FDA granted breakthrough therapy designation to the company's AVXS-101 to treat spinal muscular atrophy (SMA) type 1.

The therapy, which is in Phase I testing, uses NAV rAAV9 vector technology from RegenxBio Inc. (NASDAQ:RGNX) to deliver the survival motor neuron ( SMN) gene. AVXS-101 also has Orphan Drug designation from FDA to treat all types of SMA, and Fast Track designation to treat SMA type 1. ...