Vertex doesn’t need to do more testing for rogue gene edits from sickle cell therapy trial, FDA advisors say
Just over a month before the FDA makes a landmark decision about the first-ever therapy to use CRISPR/Cas9 gene editing, the agency is paying close attention to a hard-to-measure question: What happens if the treatment edits parts of the genetic code that it’s not supposed to?
On Tuesday, outside advisors to the agency concluded that Vertex Pharmaceuticals, which is sponsoring the potentially landmark treatment, did not need to do more testing to find potential off-target edits in current clinical trials for its experimental CRISPR-based therapy, though it did suggest long-term monitoring for such edits.
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