Aug 2, 2026
The FDA has approved Vertex Pharmaceuticals' Casgevy (exagamglogene autotemcel) for patients aged two years and older with sickle cell disease and transfusion-dependent beta thalassemia, expanding from the previous minimum age of 12 years. The approval was granted under the agency's Commissioner's National Priority Voucher program.
Intellia Therapeutics announced that its CRISPR-based gene editing treatment reduced swelling attacks in a pivotal trial for hereditary angioedema, a rare genetic disease. The results advance the CRISPR field's one-and-done treatment approach toward potential regulatory approval.