Keep an eye on CRISPR Therapeutics (CRSP).

The gene editing stock is starting to take off after a favorable US FDA panel review of the drug exa-cel for sickle-cell disease. Now, should the US FDA approve it on the panel’s vote, we could be looking at the first approved drug using gene-editing technology. 

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As noted by, “Citigroup said the overwhelming consensus from the discussion was that benefit/risk for exa-cel is exceedingly favorable, and that they expect an FDA approval with off-target monitoring language. Analysts at Truist and Mizuho also said they expect approval from the FDA, which has set a Dec. 8 target action date.”

The CRISPR/Cas9 gene-edited therapy, known as exagamglogene autotemcel or exa-cel, is currently under FDA review for SCD patients aged 12 years and older with vaso-occlusive crises, a severe characteristic of the blood disorder, added Seeking Alpha.


Ian Cooper