Novartis has reported the death of two young patients following treatment with its Zolgensma gene therapy for SMA. The fatalities were due to acute liver failure.
The deaths highlight the risks associated with one-and-done gene therapies. These therapies use inactivated viral vector delivery – in the case of Zolgensma, AAV9 vectors. But these viruses often end up in the liver, with resulting damage.
As previously posted, Astellas reported the deaths of four children following liver damage during trialling of its gene therapy for another neuromuscular condition, X-linked myotubular myopathy. That therapy used AAV8 mediated delivery.
https://www.biopharmadive.com/news/novartis-zolgensma-patient-death-liver-injury/629542/
https://www.reuters.com/business/he...a-caused-two-deaths-liver-failure-2022-08-11/
https://hotcopper.com.au/threads/th...sible-editing.6283772/page-6?post_id=56069153
Ann: PYC Therapeutics Investor Call - 18 August 2022, page-15
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