ABOVE: Red blood cells, 50x magnification
US AIR FORCE, CHRISTOPHER HUMMEL
Two companies have jointly launched a trial of an experimental CRISPR-Cas9 therapy for the blood disorder β-thalassemia, according to announcement posted Friday (August 31) on clinicaltrials.gov. Although the study itself is to be carried out in a hospital in Germany, it marks the first clinical trial of CRISPR genome-editing technology to be sponsored by US companies, Boston-based Vertex Pharmaceuticals and CRISPR Therapeutics, a Swiss biopharmaceutical with labs in Cambridge, Massachusetts.
“This is one important step of many toward bringing the promise of this new technology to patients with serious diseases like sickle cell [disease] and beta thalassemia, and we are thrilled to be at the forefront of what we believe may be a fundamental change in the treatment of disease,” Vertex spokesperson Heather Nichols says in a statement, according to STAT News.
The therapy, known as CTX001, is designed to ...
