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.

With cutting-edge technology, we are making progress in the treatment of rare and life-threatening neurological genetic diseases.Our initial gene therapy for spinal muscular atrophy (SMA) has been approved in the U.S., Japan and EU. Novartis Gene Therapies is pursuing registration in close to three dozen countries with regulatory decisions anticipated in Switzerland, Canada and Australia in late 2020 or early 2021. We are also pursuing gene therapies for Rett syndrome (AVXS-201) and a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene (AVXS-301), Friedreich’s ataxia (AVXS-401).

We are headquartered in Bannockburn, IL with another location in Deerfield, IL. Our manufacturing facilities are located in Libertyville, IL, Durham, NC and Longmont, CO. Our research site is located in San Diego, CA. European headquarters is located in Zurich, Switzerland and Novartis Pharma K.K. (“Novartis Pharma”) is located in Tokyo, Japan.

Headquarters and manufacturing around the world