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

biotechnology company Biotechnology is a multidisciplinary field that involves the integration of natural sciences and engineering sciences in order to achieve the application of organisms and parts thereof for products and services. Specialists in the field are kn ...

that develops treatments for rare neurological genetic disorders. It was founded in

Dallas Dallas () is a city in the U.S. state of Texas and the most populous city in the Dallas–Fort Worth metroplex, the List of Texas metropolitan areas, most populous metropolitan area in Texas and the Metropolitan statistical area, fourth-most ...

Texas Texas ( , ; or ) is the most populous U.S. state, state in the South Central United States, South Central region of the United States. It borders Louisiana to the east, Arkansas to the northeast, Oklahoma to the north, New Mexico to the we ...

United States The United States of America (USA), also known as the United States (U.S.) or America, is a country primarily located in North America. It is a federal republic of 50 U.S. state, states and a federal capital district, Washington, D.C. The 48 ...

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 Nationwide Children's Hospital (formerly Columbus Children's Hospital) is a nationally ranked pediatric acute care teaching hospital located in the Southern Orchards neighborhood of Columbus, Ohio. The hospital has 673 pediatric beds and is aff ...

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 ''Pari passu'' is a Latin phrase that literally means "with an equal step" or "on equal footing". It is sometimes translated as "ranking equally", "hand-in-hand", "with equal force", or "moving together", and by extension, "fairly", "without pa ...

'' 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 geneti ...

using

gene therapy Gene therapy is Health technology, medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells. The first attempt at modifying human DNA ...

. AveXis was

acquired ''Acquired'' is a podcast by Ben Gilbert and David Rosenthal about business history and strategy. As of 2024, the podcast has more than 600,000 monthly listeners. History Gilbert and Rosenthal worked together at Madrona Venture Group and ini ...

Novartis Novartis AG is a Swiss multinational corporation, multinational pharmaceutical company, pharmaceutical corporation based in Basel, Switzerland. Novartis is one of the largest pharmaceutical companies in the world and was the eighth largest by re ...

in 2018 for

USD The United States dollar (symbol: $; currency code: USD) is the official currency of the United States and several other countries. The Coinage Act of 1792 introduced the U.S. dollar at par with the Spanish silver dollar, divided it int ...

8.7 billion. In September 2020, Novartis changed the company's name to Novartis Gene Therapies. In 2019, AveXis's first gene therapy drug

onasemnogene abeparvovec Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy used to treat spinal muscular atrophy (SMA), a disease causing muscle function loss in children. It involves a one-time infusion of the medication into a vein. It ...

(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.

Controversy

Shortly after the approval, the

US Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a federal agency of the Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food ...

accused AveXis of data manipulation in their regulatory submission as well as in other regulatory submissions. After an investigation, it was alleged that Brian Kaspar and his brother Allan Kaspar, previously senior vice president of R&D, had ordered and then attempted to cover up

data manipulation Statistics, when used in a misleading fashion, can trick the casual observer into believing something other than what the data shows. That is, a misuse of statistics occurs when a statistical argument asserts a falsehood. In some cases, the misu ...

, the two were subsequently "exited" by the company

References

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