CRISPR therapy now available to toddlers
The U.S. Food and Drug Administration expanded approval for Casgevy (exagamglogene autotemcel), the CRISPR/Cas9-edited stem cell gene therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics, to include patients aged 2 years and older with sickle cell disease and transfusion-dependent beta thalassemia. Previously approved for patients 12 and older, this marks the first gene therapy available for this younger age group.
Clinical trial results support expansion
The FDA reviewed data from trials assessing efficacy and safety in patients aged 5 to 12. For sickle cell disease, 11 patients were evaluated; for transfusion-dependent beta thalassemia, 15 patients. Based on the product characteristics and available clinical data, extrapolation to children as young as 2 was supported. Vertex noted the approval could make the therapy available to approximately 5,500 additional pediatric patients in the United States.
WHO warns cancer cases could double by 2050
Separately, the World Health Organization released its Global Status Report on Cancer 2026, warning that annual new cancer cases are projected to rise from 20.6 million to nearly 35 million by 2050 unless countries take urgent action. Cancer already causes more than 26,000 deaths every day and is the world's second leading cause of death. The report highlights stark inequalities in cancer survival between high and low-income countries.