Advancing gene therapy with patients in mind

Rare diseases don't feel rare to the families devastated by them. They create daily challenges that must be faced head-on, often without the help of readily available treatment.

We are making progress in rare and life-threatening neurological genetic diseases—including spinal muscular atrophy (SMA) Type 1 and Type 2, Rett syndrome (RTT) and a genetic form of amyotrophic lateral sclerosis (ALS). Our initial product candidate, AVXS-101, is our proprietary gene therapy currently in development for the treatment of SMA Type 1, the leading genetic cause of infant mortality, and SMA Type 2.