According to this article published in BioSpace last week (as well as this article from Globe News Wire), in May of this year the FDA placed a clinical hold on an experimental trial for a treatment for sickle cell and other such diseases. The treatment was being co-developed by CRISPR Therapeutics and Vertex Pharmaceuticals, and was put on hold due to "concerns" from the FDA that needed to be reviewed before proceeding in experimental trials. The gene-editing treatment is known as CTX001, and is an ex vivo therapy for people who have inherited disorders involving hemoglobin, like sickle cell anemia or beta-thalassemia. CTX001 edits the genes through CRISPR technology to increase production of fetal hemoglobin in a patient's red blood cells. Fetal hemoglobin naturally exists in newborn babies, but sometimes carries over into adulthood, where it can provide protection for people with blood diseases. CTX001 appears to be safe because it affects only cells at the targeted site (via). The FDA did not say the specific "concerns" that they had, though it is speculated that they are being cautious about human trials because "it's a permanent alteration of a person's genetic code" (Brian Skorney). However, since CTX001 is CRISPR's "most advanced program," it seems that the FDA would rather be safe than sorry with regards to gene-editing treatments, considering potential dangers brought up regarding CRISPR technology in the past.
Ezra Lowe
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