Novartis Gene Therapies, until 2020 known as AveXis, is a

biotechnology company Biotechnology is the integration of natural sciences and engineering sciences in order to achieve the application of organisms, cells, parts thereof and molecular analogues for products and services. The term ''biotechnology'' was first used by ...

that develops treatments for rare neurological genetic disorders. It was founded in Dallas, Texas, United States in 2012 by John Carbona after reorganizing a company called BioLife Cell Bank founded by David Genecov and John Harkey. Work done at Nationwide Children's Hospital in the laboratory of Brian Kaspar was licensed to AveXis in October 2013. Unusual for the time, Nationwide Children's Hospital, in addition to upfront and milestone payments, also took an equity position in AveXis. Kaspar became paid consultant '' pari passu'' with the license agreement in 2013. The company was built specifically around a discovery of a novel method of treating

spinal muscular atrophy Spinal muscular atrophy (SMA) is a rare neuromuscular disorder that results in the loss of motor neurons and progressive muscle wasting. It is usually diagnosed in infancy or early childhood and if left untreated it is the most common genetic ...

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 Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy medication used to treat spinal muscular atrophy (SMA). It is used as a one-time intravenous injection, infusion into a vein. Onasemnogene abeparvovec works by p ...

(Zolgensma) received regulatory approval in the United States and, with a list price of USD 2.125 million per injection, became the most expensive drug in the world. In May 2020, the drug was conditionally approved in the European Union 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 AveXis of data manipulation in their regulatory submission. As a result, Brian Kaspar lost his position within the company. In September 2020, Novartis changed the company's name to Novartis Gene Therapies.

References

Pharmaceutical companies of the United States Biotechnology companies of the United States Spinal muscular atrophy {{Company-stub