- Business
- AveXis, Inc. (AVXS) is a clinical-stage gene therapy company that develops and commercializes novel treatments for patients suffering from rare and life-threatening neurological genetic diseases, including spinal muscular atrophy (SMA), Rett syndrome and a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene; its lead product candidate, AVXS-101 (now known as Zolgensma), is an investigational one-time AAV9 gene replacement therapy for SMA that has received Breakthrough Therapy designation from the U.S. FDA, PRIME designation from the European Medicines Agency and Sakigake designation in Japan, with ongoing clinical trials demonstrating compelling efficacy in SMA Type 1 patients. Founded in 2010 and headquartered in Bannockburn, Illinois, the company leverages advanced AAV9 gene delivery platforms and state-of-the-art manufacturing capabilities to address the root genetic causes of these disorders, primarily targeting pediatric and high-need patient populations in the United States and globally. In 2018, Novartis AG agreed to acquire AveXis for USD 8.7 billion in a deal unanimously approved by both boards, successfully completing the transaction through a tender offer and merger that integrated AveXis's operations, employees, gene therapy pipeline and manufacturing expertise into Novartis to accelerate global delivery of AVXS-101 and expand capabilities in neurodegenerative diseases; subsequently, in 2020, Novartis rebranded the entity as Novartis Gene Therapies, embedding it within its broader gene therapy portfolio without further standalone operational changes reported as of 2025.