Gene therapy pioneer James Wilson and University of Pennsylvania colleagues sounded an alarm Tuesday morning about the use of gene therapy to treat severe diseases like spinal muscular atrophy and Duchenne muscular dystrophy, sending a chill across the sector.
Shares of several companies inched downward as word spread about the UPenn work on Tuesday. Reached this afternoon, Wilson called for close monitoring of patients receiving high doses of a certain type of gene therapy delivered into the bloodstream.
Outside experts acknowledge that the studies were small but important. “The paper is getting the attention it deserves,” says Aravind Asokan, an associate… Read more »
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