AvaxisBio

AveXis (now Novartis Gene Therapies) is a biotechnology company that pioneered the development of gene therapies for rare and life-threatening neurological genetic diseases. The company is best known for developing Zolgensma (onasemnogene abeparvovec-xioi), the first FDA-approved systemic gene therapy for the treatment of spinal muscular atrophy (SMA). Utilizing its proprietary adeno-associated virus serotype 9 (AAV9) platform, AveXis focuses on delivering functional copies of genes to the central nervous system to address the root cause of genetic disorders. Following its $8.7 billion acquisition by Novartis in 2018, the company has expanded its research into other neurodegenerative conditions, including Rett syndrome and genetic forms of amyotrophic lateral sclerosis (ALS).