Avexis

AveXis is a biotechnology company that is focused on developing gene therapy for Spinal Muscular Atrophy (SMA) and other severe genetic diseases. The company’s initial product is currently under clinical development for SMA Type 1. SMA is a severe genetic disorder and is the leading genetic cause of infant mortality. AveXis’ treatment works to provide the critical protein that’s absence results in the breakdown of motor neurons in SMA patients.