Novartis Gene Therapies

Novartis Gene Therapies is a biotechnology business focused on developing and commercializing gene therapies for rare neurological genetic disorders. The organization originated in Dallas, Texas, in 2012 under the name AveXis. It was established by John Carbona after the reorganization of BioLife Cell Bank, a company founded by David Genecov and John Harkey. From its beginning, AveXis was built around a gene-therapy approach for spinal muscular atrophy (SMA), a severe inherited neuromuscular disease caused by insufficient survival motor neuron protein and characterized by progressive loss of motor function. A central part of the company’s early development was its relationship with Nationwide Children's Hospital and the laboratory of researcher Brian Kaspar. In October 2013, AveXis licensed work from the hospital’s laboratory concerning a method for treating SMA with gene therapy. The arrangement was notable because Nationwide Children's Hospital received not only upfront and milestone payments but also an equity interest in AveXis. Brian Kaspar became a paid consultant under the licensing relationship. This research formed the scientific foundation for the company’s lead product, onasemnogene abeparvovec, later marketed as Zolgensma. Novartis acquired AveXis in 2018 for approximately US$8.7 billion, bringing the company and its gene-therapy platform into the Novartis group. The acquisition gave Novartis a specialized rare-disease and advanced-therapy operation and positioned the business to bring its SMA treatment through regulatory review and commercialization. In 2019, onasemnogene abeparvovec received approval in the United States. The product became notable both for its one-time gene-therapy treatment model and for its US list price of US$2.125 million per injection, which made it the most expensive medicine in the world at the time. The product subsequently expanded its regulatory presence. In May 2020, it received conditional approval in the European Union for patients with SMA and a clinical diagnosis of SMA Type 1, as well as SMA patients with up to three copies of the SMN2 gene. In September 2020, Novartis renamed AveXis as Novartis Gene Therapies. The name change aligned the business more directly with its parent company while preserving its focus on gene-based treatments for serious neurological conditions. The organization’s development was also accompanied by regulatory controversy. Shortly after Zolgensma’s approval, the US Food and Drug Administration accused AveXis of data manipulation in a regulatory submission and in other submissions. Following an investigation, allegations centered on Brian Kaspar and his brother Allan Kaspar, who had served as a senior vice president of research and development. They were alleged to have ordered and attempted to conceal the manipulation, and both were subsequently removed from the company. These events became an important part of the company’s corporate and regulatory history. Novartis Gene Therapies remains associated primarily with Zolgensma and with Novartis’s broader effort to develop therapies for rare genetic and neurological diseases. Publicly available reference material identifies the company’s origin, acquisition, rebranding, lead product, and regulatory controversy, but does not provide a complete current executive roster or a comprehensive list of additional commercial products.

Novartis Gene Therapies began as AveXis, a biotechnology company founded in Dallas, Texas, in 2012 by John Carbona. Carbona formed the business after reorganizing BioLife Cell Bank, which had been founded by David Genecov and John Harkey. AveXis was created with a focused scientific and commercial objective: to develop gene-therapy treatments for rare neurological genetic diseases, especially spinal muscular atrophy. The company’s principal technology originated in research conducted at Nationwide Children's Hospital in the laboratory of Brian Kaspar. In October 2013, AveXis licensed this work, which concerned a gene-therapy method for SMA. The licensing arrangement included upfront and milestone payments and gave Nationwide Children's Hospital an equity position in AveXis, an unusual feature for a hospital research partnership at that time. Brian Kaspar also became a paid consultant under the agreement. The licensed research became the basis for the company’s lead therapy, onasemnogene abeparvovec. Novartis purchased AveXis in 2018 for approximately US$8.7 billion. The transaction incorporated AveXis into Novartis and strengthened the larger company’s presence in rare diseases and advanced gene-based medicines. In 2019, onasemnogene abeparvovec received approval in the United States under the brand name Zolgensma. It was developed as a one-time treatment for eligible patients with spinal muscular atrophy. Its US list price of US$2.125 million per injection drew extensive attention and made it the most expensive drug in the world at the time. In May 2020, Zolgensma received conditional approval in the European Union for defined SMA populations, including patients with a clinical diagnosis of SMA Type 1 and patients with up to three copies of the SMN2 gene. Later that year, in September 2020, Novartis renamed AveXis as Novartis Gene Therapies. The new name emphasized the business’s role within Novartis and its focus on gene-therapy development. The company also faced a significant regulatory controversy after the approval of Zolgensma. The US Food and Drug Administration accused AveXis of data manipulation in a regulatory submission and other submissions. An investigation alleged that Brian Kaspar and his brother Allan Kaspar, who had been a senior vice president of research and development, ordered and attempted to conceal the manipulation. Both were subsequently removed from the company. The episode affected the organization’s regulatory history and remains a major consideration in accounts of AveXis and Novartis Gene Therapies. The available reference material identifies Novartis Gene Therapies principally with Zolgensma and its SMA-focused gene-therapy platform. It does not establish a complete current leadership list, additional marketed products, or a separate public-company listing for the business.

• 2020

  Zolgensma receives conditional European Union approval

  [1]Novartis Gene Therapiesen.wikipedia.org

  The European Union conditionally approved Zolgensma for specified patients with spinal muscular atrophy.

• 2020

  AveXis becomes Novartis Gene Therapies

  [1]Novartis Gene Therapiesen.wikipedia.org

  Novartis changed the business's name to Novartis Gene Therapies in September.

• 2019

  Zolgensma is approved in the United States

  [1]Novartis Gene Therapiesen.wikipedia.org

  Onasemnogene abeparvovec became the company's first US-approved gene-therapy product for spinal muscular atrophy.

• 2018

  Novartis acquires AveXis

  [1]Novartis Gene Therapiesen.wikipedia.org

  Novartis acquired AveXis in a transaction valued at approximately US$8.7 billion.

• 2013

  SMA gene-therapy research is licensed

  [1]Novartis Gene Therapiesen.wikipedia.org

  AveXis licensed work from Brian Kaspar's laboratory at Nationwide Children's Hospital concerning gene therapy for spinal muscular atrophy.

• 2012

  AveXis is founded

  [1]Novartis Gene Therapiesen.wikipedia.org

  John Carbona founded AveXis in Dallas after reorganizing BioLife Cell Bank.

A specialized rare-disease biotechnology business within Novartis, positioned around potentially transformative, one-time gene therapies for severe inherited neurological disorders.

• Gene therapy · 2019

  Zolgensma

  Zolgensma is the brand name for onasemnogene abeparvovec, a gene-therapy treatment developed for spinal muscular atrophy. It is administered as a one-time injection and became the central commercial product associated with AveXis and later Novartis Gene Therapies. The therapy received US approval in 2019 and conditional European Union approval in May 2020 for specified SMA populations, including patients with a clinical diagnosis of Type 1 SMA or up to three copies of the SMN2 gene.

• 2019

  Regulatory data-manipulation controversy

  The US Food and Drug Administration accused AveXis of data manipulation in a regulatory submission and other submissions after Zolgensma's approval. An investigation alleged that Brian Kaspar and Allan Kaspar ordered and attempted to conceal the manipulation. Both were subsequently removed from the company.

  Source

Read more

• 2020

  Rebranding as Novartis Gene TherapiesOther

  Background

  AveXis had become a Novartis business following the 2018 acquisition.

  Action taken

  Novartis changed the company's name to Novartis Gene Therapies in September 2020.

  Aftermath

  The rebrand aligned the business more closely with Novartis and its gene-therapy focus.

  Source

• 2019

  United States launch of ZolgensmaProduct launch

  Background

  Onasemnogene abeparvovec was developed as a one-time gene-therapy treatment for spinal muscular atrophy.

  Action taken

  The product received US regulatory approval and was marketed as Zolgensma.

  Aftermath

  Its US list price of US$2.125 million per injection made it the world's most expensive drug at the time.

  US list price per injection. US$2.125 million (2019)

  Source

• 2018

  Novartis acquires AveXisM&A

  Background

  AveXis had developed a gene-therapy platform focused on spinal muscular atrophy and rare neurological genetic disorders.

  Action taken

  Novartis acquired AveXis for approximately US$8.7 billion.

  Aftermath

  The transaction brought AveXis into Novartis and provided the group with a specialized rare-disease gene-therapy business.

  Acquisition value. US$8.7 billion (2018)

  Source

1. 1Novartis Gene Therapiesen.wikipedia.org