Twenty years ago, a young man named Jesse Gelsinger was injected with a large dose of gene-shuttling viruses designed in Wilson’s lab. The experimental treatment was created to treat a rare metabolic liver disease. The goal was to deliver a working copy of the teenager’s broken gene, but the viruses threw his immune system into overdrive. Four days after being treated, Gelsinger died. It was the first public tragedy of a highly hyped field.
As details about Gelsinger’s death emerged, the entire enterprise of gene therapy began to crumble. The Gelsinger family filed a lawsuit. Investor money dried up, and start-ups shuttered. Wilson found himself at the center of multiple investigations.
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Wilson’s downsized and outcast lab kept plugging away with a newfound focus on finding safer viruses. Their work led to the discovery and dissemination of new AAVs, including one used in the recently approved gene therapy Zolgensma, which saves the lives of infants born with an otherwise fatal neurological disease.
And Zolgensma is just the beginning. By Penn’s latest count, some 42 companies are using AAVs that fall under Wilson’s patents, covering nearly 100 drug development programs.
Read full, original post: The redemption of James Wilson, gene therapy pioneer
