Approved
The first CRISPR therapy approved in the U.S. will treat sickle cell disease
U.S. patients with sickle cell disease now have a novel treatment option: the first-ever CRISPR-based therapy. On December 8, the U.S. Food and Drug Administration approved the gene-editing therapy for use in patients age 12 years and older. In addition to offering hope of relief for people with severe forms of the painful blood disorder,…
The First Crispr Medicine Is Now Approved in the US
Casgevy uses the Nobel Prize–winning technology Crispr to modify patients’ cells so that they produce healthy hemoglobin instead. The Crispr system has two parts: a protein that cuts genetic material and a guide molecule that tells it where in the genome to make the cut. To do this, a patient’s stem cells are taken out…