HEMOGLOBINOPATHIES

This study tests a new way to choose cord blood for transplant in people with blood cancers like leukemia or lymphoma. Patients get stem cells from a family member plus a cord blood unit, matched to target certain inherited traits. The goal is to find the lowest safe cord blood d…

This study tests a one-time gene therapy (CTX001) in 16 children with transfusion-dependent β-thalassemia, a genetic blood disorder requiring regular red blood cell transfusions. The treatment uses the patient's own blood stem cells, edited with CRISPR-Cas9, to produce healthy re…

This study follows 160 children and adults with beta-thalassemia or sickle cell disease who received a one-time treatment of their own CRISPR-edited stem cells (CTX001). The goal is to monitor long-term safety, including any new cancers or blood disorders, and to see how well the…

This early-phase study tests a new type of stem cell transplant for people with severe sickle cell disease who don't have a fully matched sibling donor. It uses a half-matched family donor and a milder chemotherapy regimen to prepare the body, along with removing certain immune c…

This study tests a stem cell transplant method that removes certain immune cells from the donor's blood to lower the chance of graft-versus-host disease (GvHD), a serious complication. It involves 3 patients with blood cancers or non-cancerous blood disorders who lack a perfectly…

This study tests a one-time treatment called CTX001 in 13 children with severe sickle cell disease who cannot take or did not benefit from hydroxyurea. The therapy uses the child's own blood stem cells, edited with CRISPR-Cas9 to produce healthy red blood cells. The goal is to pr…

This study tests a milder chemotherapy regimen before bone marrow or cord blood transplant in 75 children and young adults (ages 6 months to 25 years) with non-cancerous diseases like sickle cell, thalassemia, immune deficiencies, and bone marrow failure. The goal is to see if th…