The startup that manipulated data to get a miracle drug to market

Wall Street Journal

14 September 2019 - AveXis had a revolutionary gene therapy for babies with a deadly disease, and sold to Novartis for $8.7 billion. But in its hurry to get the drug approved, it made mistakes.

The startup had something incredible: a cure for babies with a deadly neurological disease. Last year, the company was snapped up by pharmaceutical giant Novartis, and by this past May, its drug was the most expensive on the market.

In just a few years, the company, AveXis, morphed from a handful of hospital-based researchers into one of the pharmaceutical industry’s most stunning success stories.

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Michael Wonder

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Michael Wonder

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US , Regulation , Gene therapy , Data