Vertex’s CRISPR treatment for sickle cell disease hits unexpected roadblock

Vertex executives warned that Casgevy, its curative treatment for sickle cell disease, would be slow to reach patients. But few expected it to be this slow.

More than two years after its approval, only about 60 patients across the U.S., Middle East, and Europe have been treated with the gene-editing therapy. Specialists at four sickle centers told STAT they’ve been surprised by one of the key stumbling blocks to faster rollout: They can’t collect enough cells to create the treatment.