First Medicine Using CRISPR Gene-Editing Gets the Go-Ahead
The Food and Drug Administration approved on Friday two breakthrough treatments using the gene-editing tool CRISPR, a Nobel Prize-winning medical technology.
The first therapy, Casgevy, was developed by Vertex Pharmaceuticals and CRISPR Therapeutics and will be used to treat sickle cell disease, a condition, according to data from the Centers for Disease Control and Prevention, that affects 100,000 Americans, a majority of whom are Black. Lyfgenia, a second gene treatment for sickle cell, was developed by bluebird bio, Inc.
“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,” Nicole Verdun, M.D., director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research, said 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.”
https://www.thedailybeast.com/first-medicine-using-crispr-gene-editing-gets-the-go-ahead
I am really chuffed about the CRISPR therapy for sickle cell. I had a patient who eventually died of the disease and I still think of him, especially now.
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