FDA expands Casgevy gene therapy to children as young as 2

The U.S. FDA on July 1 approved expanded use of Casgevy (exagamglogene autotemcel) - the world's first approved CRISPR/Cas9 gene-editing medicine, developed by Vertex Pharmaceuticals with CRISPR Therapeutics - for patients ages 2 and older with sickle cell disease or transfusion-dependent beta thalassemia, lowering the prior age floor of 12. Vertex said the expansion makes roughly 5,500 additional U.S. children newly eligible for the one-time therapy. CRISPR shares rose about 8% and Vertex about 6% on the news; CRISPR retains roughly 40% of Casgevy's economics under the partnership.