CASGEVY

Vertex presented Phase 3 data showing CASGEVY’s efficacy and safety in children 5‑11 with severe sickle cell disease (SCD) or transfusion‑dependent beta thalassemia (TDT). All 11 SCD patients remained vaso‑occlusive‑crisis‑free for ≥12 months; 12 of 13 TDT patients achieved ≥12 months transfusion independence, with one fatal conditioning event. Results mirror those in patients ≥12 years. Vertex will file global supplemental BLAs in early 2026, aiming for accelerated review, expanding the first‑in‑class gene‑editing therapy to the pediatric market.