Wikipedia
The Food and Drug Administration said on Friday that it has some safety concerns about an experimental CRISPR-based treatment for sickle cell disease, citing the methods used by its makers to evaluate the risk of inadvertently making unwanted changes to patients’ DNA.
None of the concerns raised by the FDA on Friday, however, suggest the agency is reluctant to approve the treatment, called exa-cel, which is being developed by Vertex Pharmaceuticals and CRISPR Therapeutics.
advertisement
Exa-cel, given as a one-time infusion, uses CRISPR genome-editing technology to patch up the genetic faults at the root of sickle cell disease.
Get unlimited access to award-winning journalism and exclusive events.
Subscribe Log In
Previous article：
U.K. sequencing study surfaces new findings about tumor DNA
Next article： Drug repurposing or repositioning? The language matters
Next article： Drug repurposing or repositioning? The language matters
