FDA slaps a clinical hold on sickle cell IND filed for a new gene therapy from Vertex and CRISPR Therapeutics
The FDA has slapped a clinical hold on the IND filed by Vertex $VRTX and CRISPR Therapeutics $CRSP on a new gene therapy for sickle cell disease.
The two biotechs announced an alliance on sickle cell disease late last year, co-developing CTX001, which was made with CRISPR/Cas9 tech. The drug is designed to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells.
In their statement, the companies neglected to provide much detail, saying only that the hold was put in place “pending the resolution of certain questions that will be provided by the FDA as part of its review of the IND.”
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