For the first time, the Food and Drug Administration has approved a gene-editing treatment for the purposes of fighting a human illness.
The historic decision came Friday and could help researchers eradicate sickle cell disease.
Two separate therapies have been approved for patients 12 and older who suffer from sickle cell, a blood disorder that around 100,000 Americans harbor.
"Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, 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, wrote in a statement.
"Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited," she continued.
Most of the patients who received the treatment found their severe pains resolved for at least 18 months.
The treatment was shown to have a 96.7% success rate in data presented to the FDA.
"I'm elated, excited, in awe," Jennifer Doudna of the University of California, Berkeley, who helped discover the gene-editing technique called CRISPR that is used in one of the sickle cell treatments, told NPR in an interview. "It's an exciting day and the beginning of a new day in medicine."
While not deemed a "cure" for sickle cell, the treatment edits cells to produce fetal hemoglobin, which restores normal function to red blood cells affected by the disease.
With the FDA hurdle cleared, the biggest barrier is now the price tag: Depending on which company treatment is purchased from, the price could run from $2.2 million to $3.1 million.
