Volume 94 Issue 45 | pp. 24-27
Issue Date: November 14, 2016

Gene therapy is ready for its comeback

Clinical successes, focused start-ups, and pharma’s attention push genetic remedies closer to market
Department: Business
Keywords: biologics, gene therapy, rare diseases, pharmaceuticals

Attempts to treat disease by inserting DNA into patients’ cells all but ended in 1999 after the death of an 18-year-old from a severe immune response to the virus used to deliver a corrective gene. Companies and investors left the field in droves.

Research retreated mainly to academic medical centers. Scientists—including the University of Pennsylvania’s James M. Wilson, who had been involved in the tragic trial—sought viral vectors that could . . .

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