The biggest questions facing gene therapy, the XLMTM community, and Astellas after fourth patient death
After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.
Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.
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