Dec. 6 at 8:29 PM
$CRSP VRTX just presented Casgevy data in pediatric 5-11 years old at ASH today. 4/4 SCD VOC free for at least 1 year, longest is 2 years. 6/6 TDT transfusion-free for at least 1 year, longest is 2 years. 1 TDT pt died from pneumonia following the busulfan pre-conditioning which suppresses the immune system - unfortunate circumstance probably more likely in younger children, they didnt give any other info/patient-specific susceptibilities… BEAM is working on a less harsh conditioning regimen but in adult population. VRTX will likely submit to expand label in Q1 2026, and approval will likely come latest by early Q2 because of the national priority voucher status.
