Gene therapy shows promise for sickle cell disease in small trial
Disease control
Completed
This early-phase clinical trial tested a gene therapy for adults with severe sickle cell disease. The treatment involved taking the patient's own blood stem cells, modifying them with a harmless virus to carry a healthy gene, and infusing them back after mild chemotherapy. Only 4…
Phase: PHASE1, PHASE2 • Sponsor: Donald B. Kohn, M.D. • Aim: Disease control
Last updated Jun 27, 2026 08:03 UTC