Novartis Gene Therapies

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IndustryBiotechnology, Pharmaceutical
United States
Key people
Joerg Reinhardt
ProductsOnasemnogene abeparvovec

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 Dr. David Genecov and John Harkey.[1][2] Work done at Nationwide Children's Hospital in the laboratory of Dr. Brian Kaspar was licensed to AveXis in October 2013. Unusual for the time, Nationwide Children's in addition to upfront and milestone payments, also took an equity position in AveXis.[3] Dr. Kaspar became paid consultant pari passu with the license agreement in 2013.[4] Specifically 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 has a list price of USD 2.125 million per injection, becoming the most expensive drug in the world.[15] 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.[16]

Shortly after the approval, the US Food and Drug Administration accused the company of data manipulation in their regulatory submission.[17] As a result, Brian Kaspar lost his position within the company.[18]

In September 2020, AveXis changed its name to Novartis Gene Therapies.[19][20]


  1. ^ Moore, Charles SMA News Today May 7, 2015
  2. ^ Speights, Keith The Motley Fool, May 30, 2019 Bothered by the Price Tag of a $2.1 Million Drug? Read This Before Complaining Too Much
  3. ^ Ghose, Carrie, Columbus Business First Apr 9, 2018://
  4. ^ AveXis- BioLife Licenses Spinal Muscular Atrophy (SMA) Patent Portfolio from Nationwide Childrens Hospital and The Ohio State University,
  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. 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. ^ "$2.1m Novartis gene therapy to become world's most expensive drug". The Guardian. 24 May 2019. Retrieved 3 July 2019.
  15. ^ Reuters (2019-05-25). "$2.1m Novartis gene therapy to become world's most expensive drug". The Guardian. ISSN 0261-3077. Retrieved 2019-08-14.
  16. ^ AveXis receives EC approval and activates “Day One” access program for Zolgensma®, the only gene therapy for spinal muscular atrophy (SMA), PM Novartis May 19, 2020; retrieved May 20, 2020 Public Domain This article incorporates text from this source, which is in the public domain.
  17. ^ Commissioner, Office of the (2019-08-06). "Statement on data accuracy issues with recently approved gene therapy". FDA. Retrieved 2019-08-14.
  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. Retrieved 2020-10-16.
  20. ^ Melamed, David. "AveXis, Developer of Zolgensma, Now Known as Novartis Gene Therapies". Retrieved 2020-10-16.