Novartis Gene Therapies

AveXis is a biotechnology company that develops treatments for rare neurological genetic disorders. It was founded in Dallas, Texas in 2012 by John Carbona after reorganizing a company called BioLife Cell Bank founded by Dr. David Genecov and John Harkey. Work done at Nationwide Childrens Hospital in the laboratory of Dr. Brian Kaspar was licensed to AveXis in October 2013. Unusual for the time, Nationwide Childrens in addition to upfront and milestone payments, also took an equity position in AveXis. Dr. Kaspar became paid consultant pari passu with the license agreement in 2013. Specifically a discovery of a novel method of treating spinal muscular atrophy using gene therapy.
AveXis was acquired by Novartis in 2018 for USD 8.7 billion.
In 2019, Avexis's first gene therapy drug onasemnogene abeparvovec (Zolgensma®) received regulatory approval in the United States and has a list price of USD 2.125 million per injection, becoming the most expensive drug in the world. In May 2020, Zolgensma® is conditionally approved in Europe for the treatment of patients with spinal muscular atrophy (SMA) and a clinical diagnosis of SMA Type 1; or SMA patients with up to three copies of the SMN2 gene.
Shortly after the approval, the US Food and Drug Administration accused the company of data manipulation in their regulatory submission. As a result, Brian Kaspar lost his position within the company.
In September 2020, AveXis changed its name to Novartis Gene Therapies.