Based in Chicago, Illinois, AveXis is a clinical-stage gene therapy company developing innovative treatments for rare and life-threatening genetic diseases. Our gene therapy product candidate, scAAV9.CB.SMN (AVXS 101), is currently in a Phase 1 clinical trial in the United States for the treatment of spinal muscular atrophy (SMA) type 1. SMA is the most common cause of genetic infant mortality and the second most common autosomal recessive disease. No treatment is currently approved for the treatment of SMA in the United States, Europe or other geography. The SMA program has received Orphan Drug designation in both the United States and Europe.
AveXis is a rapidly growing leader among gene therapy biotech companies and continues to establish relationships with academics, biotechnology and pharmaceutical companies, and healthcare investors.