The Top Line

August 19, 2022


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Novartis has recorded two deaths after treatment with its gene therapy Zolgensma. The treatment is for spinal muscular atrophy. Two children in Russia and Kazakhstan died about five to six weeks after receiving Zolgensma. Both patients died of acute liver failure, a known side effect of Zolgensma. In today's episode, we will discuss these deaths and the gene therapy’s safety.

AstraZeneca may be a major oncology player, but the company has been noticeably absent in the first wave of CAR-T therapies. All that may be about to change. We chat with Dave Fredrickson, from the Big Pharma's oncology unit, about how the company is working on the next generation of these cancer therapies.

To learn more about the topics in this episode: 

  • 2 deaths after Novartis' Zolgensma put gene therapy's liver safety in the spotlight once again 
  • Late to the CAR-T game, AstraZeneca quietly works on its own off-the-shelf therapies 
  • UPDATED: Bluebird bio's $2.8M gene therapy Zynteglo wins FDA backing. Will its US launch take flight?
  • Hear ye, hear ye: FDA finalizes OTC hearing aid rule to amp up access, drive down costs 
  • Sanofi scraps oral SERD strategy after 2nd failure in breast cancer 
  • Are SERDs dead? 'Good question,' says Sanofi R&D chief, after French pharma's late-stage pipeline takes a beating 
  • After billions in legal costs, Johnson & Johnson plans to halt talc sales worldwide
  • AstraZeneca heads to court to contest former exec's move to crosstown rival GSK: report
  • CPAP machine competitor ResMed nears $3.6B revenue in first year of Philips' ventilator recall 
  • Merck bets big on circular RNA, paying $150M and dangling $3.5B in biobucks to work with Orna 
  • AZ, Daiichi Sankyo's Enhertu breaks more ground, nabbing fast FDA nod in HER2-mutant lung cancer 
  • We're looking for 2022's Fiercest Women in Life Sciences 
  • The Top Line is produced by senior multimedia producer Teresa Carey with managing editor Querida Anderson and senior editors Annalee Armstrong, Ben Adams, Conor Hale and Eric Sagonowsky. The sound engineer is Caleb Hodgson. The stories are by all our “Fierce” journalists. Like and subscribe wherever you listen to your podcasts.

    See omnystudio.com/listener for privacy information.

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