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Novartis Gene Therapies

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Novartis Gene Therapies
ProductsOnasemnogene abeparvovec

Novartis Gene Therapies, until 2020 known as AveXis, is a biotechnology company 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.[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.[3] Kaspar became paid consultant pari passu with the license agreement in 2013.[4] The company was built specifically around a discovery of a novel method of treating spinal muscular atrophy using gene therapy.[5] AveXis was acquired by Novartis in 2018 for USD 8.7 billion.[6][7][8][9][10][11][12]

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

Shortly after the approval, the US Food and Drug Administration accused AveXis of data manipulation in their regulatory submission as well as in other regulatory submissions.[16] 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, the two were subsequently "exited" by the company[17][18]

In September 2020, Novartis changed the company's name to Novartis Gene Therapies.[19][20]


  1. ^ Moore, Charles (7 May 2015). "John Carbona Steps Down as AveXis CEO; Dallas Based AveXis Inc. Announces Management Succession Plan - SMA News Today". Retrieved 2022-06-15.
  2. ^ Speights, Keith (2019-05-30). "Bothered by the Price Tag of a $2.1 Million Drug? Read This Before Complaining Too Much". The Motley Fool. Retrieved 2022-06-15.
  3. ^ Ghose, Carrie (April 9, 2018). "Nationwide Children's sold stock, won't get $100M windfall in $8.7B acquisition of gene therapy spinoff". Columbus Business First. Retrieved 2022-06-15.
  4. ^ "AveXis- BioLife Licenses Spinal Muscular Atrophy (SMA) Patent Portfolio from Nationwide Childrens Hospital and The Ohio State University". Nationwide Childrens Hospital. Retrieved 2022-06-15.
  5. ^ "Zolgensma's Journey from Lab Idea to Gene Therapy for SMA". SMA News Today. 2019-05-27. Retrieved 2019-08-14.
  6. ^ Herper, Mathew (19 December 2018). "A Year In, Novartis' Boss Faces The World". Forbes. Retrieved 10 January 2018.
  7. ^ Delclaux, Alberto (9 April 2018). "Novartis Bets $8.7 Billion on Gene-Therapy Company". The Wall Street Journal. Retrieved 10 January 2018.
  8. ^ Rose, Marla (9 April 2018). "Gene-therapy company with research ties to Columbus sold to Novartis for $8.7 billion". The Columbus Dispatch. Archived from the original on 26 April 2018. Retrieved 10 January 2018.
  9. ^ Miller, John (9 April 2018). "Novartis bets big on gene therapy with $8.7 billion AveXis deal". Reuters. Retrieved 10 January 2018.
  10. ^ Kresge, Naomi (9 April 2018). "Novartis Wager on AveXis Shows Rare Diseases Command Mega Prices". Bloomberg News. Retrieved 10 January 2018.
  11. ^ Kresge, Naomi (9 April 2018). "Novartis CEO Spurs Rare-Disease Shift With $8.7 Billion Deal". Bloomberg News. Retrieved 10 January 2018.
  12. ^ Woodyard, Chris (9 April 2018). "Novartis buying AveXis for $8.7B in big gene therapy bet". USA Today. Retrieved 10 January 2018.
  13. ^ Kaiser, Kaiser (1 November 2017). "Gene therapy's new hope: A neuron-targeting virus is saving infant lives". Science. Retrieved 10 January 2018.
  14. ^ a b "$2.1m Novartis gene therapy to become world's most expensive drug". The Guardian. 24 May 2019. Retrieved 3 July 2019.
  15. ^ AveXis receives EC approval and activates “Day One” access program for Zolgensma®, the only gene therapy for spinal muscular atrophy (SMA) Archived 2020-05-29 at the Wayback Machine, PM Novartis May 19, 2020; retrieved May 20, 2020 Public Domain This article incorporates text from this source, which is in the public domain.
  16. ^ Commissioner, Office of the (2019-08-06). "Statement on data accuracy issues with recently approved gene therapy". FDA. Retrieved 2019-08-14.
  17. ^ Palmer, Eric (September 24, 2019). "Novartis to put AveXis into protective custody after data manipulation scandal". Fierce Biotech. Retrieved May 12, 2024.{{cite news}}: CS1 maint: url-status (link)
  18. ^ "Novartis replaces top scientists at Avexis after drug data manipulated". Reuters. 2019-08-14. Retrieved 2019-08-14.
  19. ^ "With new AveXis name, Novartis spotlights marquee role for gene therapy business". FiercePharma. 4 September 2020. Retrieved 2020-10-16.
  20. ^ Melamed, David (14 September 2020). "AveXis, Developer of Zolgensma, Now Known as Novartis Gene Therapies". Retrieved 2020-10-16.