Xconomy: Stop the Bleeding: Can Gene Therapy Finally Cure Hemophilia?

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Summary

While nearly all for-profit activity in gene therapy ground to a halt after the bubble crash and trial deaths, High, Wilson, and others not only kept the field afloat, but helped make the advances that have led to the current hemophilia race.Shaken up and in no position to compete for NIH grant money after the Gelsinger fiasco, he went to an old mentor, Tachi Yamada, then the chief scientific officer of SmithKline-Beecham (now GlaxoSmithKline).Wilson got about $40 million from the big British drug maker over the next several years and went “subterranean.” He eschewed scientific lectures, he stayed away from awards banquets, and he avoided the press.High recruited folks from Avigen, and one of them, Fraser Wright (now Spark’s chief technology officer), applied for and won an NIH contract to be the only federally-funded AAV manufacturing facility in the country.Another was a group split between St. Jude Children’s Research Hospital in Memphis, TN, and University College London who were intrigued by the UPenn dog study and wanted to try AAV8 for hemophilia in humans.

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