AveXis is dedicated to developing and commercializing gene therapies for patients and families devastated by rare and life-threatening neurological genetic diseases.

Our initial proprietary investigational gene replacement therapy,
ZOLGENSMA® (onasemnogene abeparvovec-xioi, AVXS-101),* is in development for the treatment of spinal muscular atrophy (SMA), a rare and devastating genetic disease that leads to progressive muscle weakness, paralysis and, when left untreated in its most severe form (Type 1), death.

*The brand name ZOLGENSMA® (onasemnogene abeparvovec-xioi) has been provisionally approved by the FDA for the investigational product AVXS-101, but the product itself has not received marketing authorization or Biologics License Application approval from any regulatory authorities.