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Novartis dumps Rett gene therapy caught up in Zolgensma scandal in cull of pipeline prospects

October 26, 2021

Novartis has scrapped work on its Rett syndrome gene therapy AVXS-201 after finding the data don’t support further development. The action follows Novartis’ decision to gather extra data on the candidate in response to data manipulation during development of Zolgensma.

When the Zolgensma scandal hit in 2019, Novartis decided to review data quality and compliance for preclinical work on AVXS-201 and to repeat and add pivotal preclinical studies and quality controls. The extra work set back plans to get AVXS-201 into the clinic. In August 2020, Novartis said it had finished most of the preclinical studies, putting it a little behind the schedule it set out the prior year but still on track to file an IND and get AVXS-201 into the clinic.

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