In preparation for gene therapy, chemotherapy conditioning is given to patients; ninety-five percent of those patients had a grade 3 or 4 toxicity from the conditioning.

Eighty-six percent developed mucositis.2 The cause was the busulfan that prepared them for a curative treatment for sickle cell disease.

Curative cell and gene therapies are gated by a fifty-year-old chemotherapy regimen. Removing that gate opens them to everyone currently too old or too fragile to qualify.

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