Novartis Gene Therapies, until 2020 known as AveXis, is a biotechnology company that develops treatments for rare neurological genetic disorders.

[1][2] 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.

[4] The company was built specifically around a discovery of a novel method of treating spinal muscular atrophy using gene therapy.

[13][14] In 2019, AveXis's first gene therapy drug onasemnogene abeparvovec (Zolgensma) received regulatory approval in the United States[15][16] and, with a list price of USD 2.125 million per injection, became the most expensive drug in the world.