Cell, Gene, and CAR-T Therapy Milestones

Zanvastro is the first FDA-approved disease-modifying treatment for Alexander disease, FAYUVI is approved for pediatric MPS IIIA, and CASGEVY is available to children aged two and older with sickle cell disease or transfusion-dependent beta thalassemia. Sickle-cell gene therapy reviews emphasize that conditioning, specialized delivery, monitoring, reimbursement, and family counseling limit access despite transformative efficacy. PCSK9 editing, cilta-cel, anti-CD7 CAR-T, and CD33-deleted donor cells continue to show potential alongside major uncertainty about long-term benefit and toxicity.