In a historic first, the Food and Drug Administration (FDA) has approved America's first gene therapies for sickle-cell disease (SCD), one of which uses the gene-editing tool CRISPR.
"Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need [for better, long-lasting treatments], and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today," Dr. Nicole Verdun, director of the Office of Therapeutic Products within the FDA's Center for Biologics Evaluation and Research, said in a statement released Friday (Dec. 8).
