The first Investigational New Drug (IND) application for base-editing technology has been cleared by the Food and Drug Administration. BEAM-101, developed by Beam Therapeutics, is an ex vivo base-editing product candidate, meaning that it uses a modified form of CRISPR capable of making single base changes without double-stranded DNA cleavage.Continue reading
There is one more hopeful story from NPR. It involves a woman with a congenital eye disorder who volunteered to have her retina edited. A few months ago, it was a man suffering from a rare liver disease. The first of all, as you probably know, was a woman struggling with sickle cell disease. Don’t miss their CRISPR stories!
The first human CRISPR trial approved in the United States is finally recruiting the first patient. In the meantime trials have grown to a dozen in China, considering those revealed by the Wall Street Journal inquiry besides the NIH database (check also this npr article for further details). Over 80 Chinese patients are already receiving a CRISPR-based treatment, while US researchers cautiously plan to test the safety of their experimental therapy on a single subject, and, if everything goes right, two more patients will be treated a month later. Is the West losing its genome-editing edge to Beijing? Continue reading